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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 1.0-999.0, Mitochondrial Diseases Patients with mitochondrial disease (clinical and histological criteria) Or Patients for whom an exome analysis has been performed, who are carriers of a pathogenic or probably pathogenic variant, in a heterozygous state, in an autosomal recessive transmission gene strongly candidate in view of the patient's clinic Or patients for whom exome analysis did not reveal any pathogenic variant explaining the phenotype People hospitalized without consent Phenotype not suggestive of mitochondrial disease
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Male or female ≥ 18 years of age. 2. Myasthenia Gravis Foundation of America (MGFA) Class II-IVa and likely not in need of a respirator for the duration of the study as judged by the Investigator. 3. QMG score ≥12 at Screening and Baseline. Other, more specific are defined in the protocol Use of rituximab, belimumab, eculizumab or any monoclonal antibody for immunomodulation within 6 months prior to first dosing. 2. Immunoglobulins given by SC, IV (IVIG), or intramuscular route, or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening. 3. Thymectomy performed < 12 months prior to screening. 4. Total IgG level <6 g/L (at screening). 5. Absolute neutrophil count <1500 cells/mm3(at screening). Other, more specific are defined in the protocol
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Breast Cancer Joint Pain Women with histologically confirmed primary invasive adenocarcinoma of the breast, stages I-IIIA Estrogen-receptor positive (ER+) and/or progesterone-receptor positive (PR+) breast cancer Completion of definitive surgery with mastectomy or breast conserving therapy Postmenopausal (no menses >= 12 months) or on ovarian suppression in order to take AIs Currently taking an Food and Drug Administration (FDA) approved third-generation aromatase inhibitor (e.g., anastrozole [Arimidex], letrozole [Femara], or exemestane [Aromasin]) for >= 90 days prior to registration with plans to continue for >= 90 days after registration Clinical symptoms of joint pain for at least 3 months prior to study entry that started or increased with AI therapy with Brief Pain Inventory (BPI) Worst Pain score >= 4 (verbal response to BPI question 3 regarding the worst pain in the past 24 hours as 0 "no pain" to 10 "pain as bad as you can imagine") Prior malignancy =< 5 years except adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, ductal carcinoma in situ of the breast or adequately treated stage I or II cancer from which the patient is currently in complete remission History of a bleeding tendency or current use of coumadin or other anticoagulants Current or previous history of anemia Current autoimmune, liver, hematopoietic, cardiac, or renal disease Current viral, bacterial, atypical or fungal infections of any organ system Concurrent use of immunosuppressant medications Concurrent use of medications known to inhibit or induce hepatic enzyme CYP 3A4 (e.g. ketoconazole, macrolide antibiotics, barbiturates) Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situation that would limit compliance with study requirements Bone fracture or surgery of the affected joints, within 180 days of study entry Medical therapy, alternative therapy, or physical therapy for joint pain/stiffness =< 30 days of study entry
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 12.0-17.0, Non-Alcoholic Fatty Liver Disease NAFLD Pediatric NAFLD For clinical referral to screening visit: 1. Age: 12 to <20 years old 2. Diagnosis of Obesity: BMI-percentile ≥95th (using age and sex based Center for Disease Control definitions) or BMI ≥30 kg/m2 3. Elevated alanine aminotransferase (ALT) more than twice the upper limit of normal by gender (≥44 U/L for girls, ≥50 U/L for boys) within 3 months prior to screening (used for historic ALT value) OR diagnosis of NAFLD from ultrasound, MRI, or participants with biopsy-proven NASH within 12 moths of screening 4. History of lifestyle modification to treat obesity or NAFLD To be obtained at screening visit: 1. Confirmation of Obesity 2. Tanner stage 2 3. Normal fasting glucose tolerance (fasting blood glucose <100 mg/dL) 4. If Screening ALT is used as [if > 2x historic ALT value (historical value obtained clinically within 12 months of screening visit), repeated after 4 weeks [unable to randomize until completed]]. If the repeat ALT is more than 50% increased or decreased over the screening ALT, a third ALT should be obtained. If a third ALT is not within 50% of the previous value, then the subject is ineligible but may be screened at a later date. If ALT is not used An ultrasound will be done to diagnose NAFLD if the diagnosis has not previously been made by ultrasound, MRI or biopsy A MRI-derived HFF ≥ 5.5% 5. Willingness to adhere to lifestyle considerations throughout the study ALT > 250U/L at screening 2. History of significant alcohol intake or current use 3. Impaired fasting glucose (>100 mg/dL) 4. Diabetes (type 1 or 2) 5. Current or recent (<6 months prior to enrollment) use of weight loss medication(s) 6. Vitamin E supplementation 7. Previous bariatric surgery 8. Use of metformin 9. Prior use of empagliflozin 10. Lower limb infection/ulceration within 3 months of screening 11. Metal or magnetic implants, devices or objects inside of or on the body, which are not MRI compatible 12. Structural and functional urogenital abnormalities, that predispose for urogenital infections 13. Recent initiation (<3 months prior to enrollment) of anti-hypertensive or lipid medication(s) 14. Major psychiatric disorder 15. Current pregnancy or plans to become pregnant.Females unwilling to be tested for pregnancy. Females will be tested for pregnancy. Females who are sexually active and not protected by an effective method of birth control (e.g. UID or medication or patch) 16. Tobacco use 17. Significant liver dysfunction (levels >5 times the upper limit of normal (ULN)): ALT (ULN = 50 U/L) AST (ULN = 48 U/L) GGT (ULN = 48 U/L) ALP (ULN = 115 U/L) 18. Platelets < 150,000 cells/mm3 19. Total bilirubin 1.3 mg/dL 20. INR 1.3 21. Albumin <3.2 g/dL 22. Gilbert's Syndrome 23. Any known causes of liver disease (except NAFLD and NASH) 24. Significant renal dysfunction (estimated glomerular filtration rate [eGFR] < 90 mL/min/1.73 m2), 25. Diagnosed monogenic obesity 26. History of cancer 27. Untreated thyroid disorder 28. History of decompensation events (ascites, variceal bleeding, hepatic encephalopathy, or hepatocellular carcinoma) 29. Current or recent (<6 months prior to enrollment) use of medication(s) associated with weight gain (e.g. atypical anti-psychotics)
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-65.0, Acute Coronary Syndrome Healthy male subjects, or female subjects not of childbearing potential (either surgically sterile or post-menopausal)] Age between 18 and 65 years inclusive Non-smokers Body mass index (BMI) between 18 and 28 kg/m2 inclusive, with a body weight between 60-100 kg In good health as determined by a medical history, physical examination, vital signs and clinical laboratory test results, including renal and liver function, and full blood count Provision of informed consent before any trial-related activity Any history of cancer, diabetes or, in the opinion of the investigator, clinically-significant cardiovascular, respiratory, metabolic, renal, hepatic, gastrointestinal, haematological, dermatological, neurological, psychiatric or other major disorders Any history of either significant multiple drug allergies or known allergy to the study drugs or any medicine chemically related to the study drugs A clinically-significant illness within 4 weeks of randomisation Any clinically-significant abnormal laboratory test results at screening in the opinion of the investigator A supine blood pressure at screening, after resting for 5 minutes, higher than 150/90 mmHg or lower than 105/65 mmHg A supine heart rate at screening, after resting for 5 minutes, outside the range of 50-100 beats/min Receipt of any prescribed or over-the-counter systemic or topical medication within 48 hours prior to the start of dosing Planned or expected requirement, during the next 3 months (at randomisation, or 3 weeks at the start of period 2), for any systemic or topical prescribed drug, or for systemic or topical over-the-counter NSAID, corticosteroid, anthihistamine or any other drug that could affect inflammation, thrombosis or haemostasis in the opinion of the investigator Receipt of an investigational medicinal product within the previous four months (new chemical entity) or three months (licensed product) or subjects who have received a vaccine within three months preceding the start of dosing. When reconfirming at the start of period 2, receipt of aspirin, ticagrelor or endotoxin during period 1 of this study will not be counted for this purpose Any donation of blood or plasma in the month preceding the start of dosing
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-999.0, HIV-1 For HIV+ women who give consent, the measurement of their HIV-1 viral load and the detection of HIV-1 DNA in their children will be assessed. A mother/infant pair will be included in the Phase III trial if the infant Is a singleton Is breastfed at 2 months and the mother intends to continue breastfeeding for at least 4 months (until her child is 6 months old) Has a negative HIV-1 PCR POC test at 2 months of age Has a mother who Is the accompanying person to visit 2 of the EPI Is 15 years of age or older (in Zambia) and 20 years of age or older (in Burkina Faso) or If between 15 and 19 years of age (inclusive) in Burkina Faso, and is accompanied by a referent adult of her choice representing her interests and the interests of the child (parent, family member or guardian, member of an association, etc.) Has been confirmed to be infected with HIV-1 (with or without HIV-2) Has signed the consent form to participate For the mother in Zambia, the consent must be signed by herself and a witness; For the mother in Burkina Faso, the consent must be signed by herself and a witness (if illiterate) and/or a referent adult (if under 20 years of age in Burkina Faso). For the child in Zambia, the consent must be signed by the mother. For the child in Burkina Faso, the consent must be signed by the mother and/or a referent adult (if under 20 years of age in Burkina Faso. In Burkina Faso, both parents need to sign the consent unless the mother exercises sole parental authority or if obtaining the father's consent is likely to endanger the mother and her child. In Zambia, the mother exercises sole parental authority A mother-child couple will not be included if the child Has clinical symptoms or biological abnormalities of DAIDS classification 3 or 4 for adverse events on the day of Has a severe congenital malformation Has a known allergy to the study drug or its components Takes emtricitabine concomitantly Has a mother who Lives outside the study area or intending to move from the area within the next 12 months Is participating in another clinical trial
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Critical Illness Polyneuropathy Critical Illness Myopathy Weakness Due to Disease Neuromuscular Blockade Adult patients (18-year-old and older ones) hospitalised in ICU. In order to realise the first aim of the study (to estimate the prevalence of NMJ disorders acquired during critical illnesses using SF-EMG), patients with a pathologic PENT will be evaluated. In order to realise the second aim of the study (to assess the reliability of TOF in critically ill patients. The study will evaluate patients with critical illnesses hospitalised in the General Intensive Care Unit), patients who receive NMBAs will be evaluated; patients who do not receive NMBAs will also be evaluated, as a control group patients with a terminal disease patients with an ICU-length-of-staying perspective less than 72 hours patients with a chronic disease associated with known neuropathy or myopathy patients treated with drugs that may cause neurotoxicity and myotoxicity patients with primary or secondary acute Peripheral Nervous System (PNS) lesions or muscle lesions patients with prosthesis, wounds or other conditions that prevent electrophysiological testing
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Richter Syndrome Entry for randomised trial component (standard of care and experimental arms): for the randomised trial component Suitable for anthracycline-containing chemo-immunotherapy Patients with CLL and newly diagnosed biopsy proven DLBCL-type RS ECOG performance status of 0, 1, 2 or 3 Age 16 years and over Signed written informed consent prior to performing any study-specific procedures for the randomised trial component Prior therapy with CHOP or any anthracycline containing treatment at any time prior to randomisation. (Please note that pre-treatment with prednisolone up to 2mg/kg is allowed for up to 14 days prior to the start of treatment) Ibrutinib-exposed CLL patients who have been newly diagnosed with RS within four weeks of their last dose of ibrutinib. (Ibrutinib-exposed CLL patients who discontinue ibrutinib due to toxicity or progressive CLL and later (more than four weeks) develop RS are not excluded from the randomised trial component) Previous acalabrutinib exposure. (Prior exposure to other Bruton tyrosine kinase (BTK), phosphoinositide-3-kinase (PI3K), or BCL-2 inhibitors is permitted, with the exception of patients who have progressed on ibrutinib see criterion above) Known central nervous system (CNS) involvement of CLL or DLBCL Any other active malignancy that requires active treatment, with the exception of basal cell carcinoma, in-situ cervical cancer, and non-invasive squamous cell carcinoma of the skin Chronic or ongoing active infectious disease requiring systemic treatment such as, but not limited to, chronic renal infection, chronic chest infection with bronchiectasis, tuberculosis, and active hepatitis Positive serology for Hepatitis B (HB) defined as a positive test for HB surface antigen (HBsAg). In addition, if negative for HBsAg but HB core antibody (HBcAb) positive (regardless of HBsAb status), a HBV deoxyribonucleic acid (DNA) test will be performed and if positive the patient will be excluded Known human immunodeficiency virus (HIV) positive
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-80.0, Myasthenia Gravis Able to provide signed informed consent Able to read and understand Danish or English Diagnosed with mild to moderate myasthenia gravis (I-IV on the Myasthenia Gravis Foundation of American Clinical Classification, MGFA) Documented history of acetylcholine receptor (AChR) or Muscle Specific Kinase (MuSK) antibody positive, or abnormal repetitive nerve stimulation testing (decrement > 10%) on EMG or abnormal single fiber EMG (conduction block or jitter) or based on their clinical history and symptom improvement with acethylcholinesterase inhibitors If the patient is on oral corticosteroids, the dose must be stable for at least 1 month prior to inclusion If the patient is on cholinesterase inhibitors the dose must be stable 2 weeks prior to inclusion MGFA grade V disease Other disorders that are not related to MG, or drugs, that interfere with muscle strength, balance and fatigue Serious medical illness (e.g. uncontrolled insulin dependent diabetes mellitus, symptomatic coronary artery disease, and cancer) Dementia or pregnancy Unspecified reasons judged by the investigator
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Autoimmune Diseases autoimmune or infectious disease
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.083-18.0, Kawasaki Disease Appendicitis Patients with Kawasaki disease Acute abdomen requiring surgery Patients with chronic gastrointestinal disease Incomplete data on the charts
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-80.0, Myasthenia Gravis Able to provide signed informed consent Able to read and understand Danish or English Diagnosis of mild to moderate MG (Able to provide signed informed consent Able to read and understand Danish or English Diagnosed with generalized mild to moderate myasthenia gravis (I-IV on the Myasthenia Gravis Foundation of American Clinical Classification, MGFA) Documented history of acetylcholine receptor (AChR) or Muscle Specific Kinase (MuSK) antibody positive, or abnormal repetitive nerve stimulation testing (decrement > 10%) on electromyography (EMG) or abnormal single fiber EMG (conduction block or jitter) or based on their clinical history and symptom improvement with acetylcholinesterase inhibitors Ability to walk > 60 meters in a 6MWT MGFA grade V disease Other disorders that are not related to MG, or drugs, that interfere with muscle strength, walking ability, balance and fatigue (e.g. heart failure) Serious medical illness (e.g. uncontrolled insulin dependent diabetes mellitus, symptomatic coronary artery disease, and cancer) Dementia or pregnancy Unspecified reasons judged by the investigator
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 12.0-999.0, Ann Arbor Stage III Hodgkin Lymphoma Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma Ann Arbor Stage IIIA Hodgkin Lymphoma Ann Arbor Stage IIIB Hodgkin Lymphoma Ann Arbor Stage IV Hodgkin Lymphoma Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma Ann Arbor Stage IVA Hodgkin Lymphoma Ann Arbor Stage IVB Hodgkin Lymphoma Classic Hodgkin Lymphoma Lymphocyte-Rich Classic Hodgkin Lymphoma All patients must have histologically confirmed newly diagnosed, previously untreated stage III or IV classical Hodgkin lymphoma (nodular sclerosing, mixed cellularity, lymphocyte-rich, or lymphocyte-depleted, or not otherwise specified [NOS]). Nodular lymphocyte predominant Hodgkin lymphoma is not eligible Patients must have bidimensionally measurable disease (at least one lesion with longest diameter >= 1.5 cm) documented on the Lymphoma Baseline Tumor Assessment Form in Rave Patients must have a whole body or limited whole body PET-CT scan performed within 42 days prior to registration. (A contrast-enhanced [diagnostic] CT, MRI or MR-PET is acceptable in event that PET-CT is contra-indicated, however if it is later possible to administer a PET-CT, then PET-CT is strongly preferred for the interim scan (after cycle 2) (if performed) and the EOT assessment. Otherwise, if PET-CT is not subsequently possible, then the same modality as baseline must be used throughout the trial.) NOTE: All images from PET-CT, CT, MRI or MR-PET scans performed as standard of care to assess disease (within 42 days prior to registration) must be submitted and associated radiology reports must be submitted Patients must not have received any prior chemotherapy, radiation, or antibody-based treatment for classical Hodgkin lymphoma. Steroid pre-treatment is permitted Patients must not have had prior solid organ transplant Patients must not have had prior allogeneic stem cell transplantation Patients must not have received a live vaccine within 30 days prior to planned day 1 of protocol therapy (e.g. measles, mumps, rubella, varicella, yellow fever, rabies, Bacillus Calmette-Guerin [BCG], oral polio vaccine, and oral typhoid) At registration, investigator must declare intent-to-treat with residual PET radiation therapy (residual PET RT RPRT) to be administered after patient completes 6 cycles of therapy if, after end of treatment, the patient meets specified for receiving RT). Patients will be stratified by investigator's intent-to-treat with residual PET RT All pediatric patients (< 18 years of age) will be considered intent-to-treat with Residual PET RT at time of registration
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Generalized myasthenia gravis (MGFA IIa-IVb) at screening, verified by ≥ 1 of the following: 1) AchR-antibodies in medical history, 2) Abnormal decrement on repetitive nerve stimulation in medical history Disease duration of ≥ 1 year Stable dose of antimyasthenic medications at screening Residual symptoms with a MG-QOL15 score of ≥ 10 Age ≥ 18 years Ability to understand the requirements of the trial and provide written, informed consent Evidence of malignancy ≤ 3 years prior to screening, unless deemed completely cured Thymectomy ≤ 6 months prior to screening Impending MG crisis or respiratory insufficiency Worsening of MG symptoms due to other diseases or medications (e.g. infection, beta-blockers, aminoglycosides, etc.) Other factor(s) or medical condition(s) that may explain residual symptoms Pregnancy or breast-feeding Treatment with beta-agonists Uncontrolled diabetes Ischemic Heart Disease, Cardiac Arrhythmia or Heart Failure (including hypertrophic cardiomyopathy) Uncontrolled Hypertension (≥ 160/110)
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 16.0-999.0, Thalassemia Subject Thalassemia patients with serum ferritin ≥ 500 µg/L Patients aged 16 and above HB≥80 g/L before administration Voluntarily participate in the experiment, and the process of obtaining informed consent met the requirements of GCP. Subject Hepatitis B surface antigen positive, hepatitis B core antibody positive and HBV-DNA positive, hepatitis C anti-HCV positive, HIV positive, Treponema pallidum positive History of active digestive tract diseases (including gastric ulcer, duodenal ulcer, gastroesophageal varices, ulcerative colitis, Crohn's disease, digestive tract tumors, familial genetic polyps), history of digestive tract perforation, history of digestive tract surgery and influence on drug absorption, and other investigators believe that patients with potential intestinal complications Liver dysfunction (ALT or AST > 2.5×ULN); or renal dysfunction (serum creatinine > 1.5×ULN) Uncontrolled active infections Patients currently taking CYP3A strong inducer or inhibitor drugs or drugs that may prolong the QT interval without temporary suspension of use or temporary substitution of the said drugs Allergic constitution: allergic to or with contraindication of main ingredients or excipients of CN128 tablets (microcrystalline cellulose, cross-linked sodium carboxymethyl cellulose, silica, sodium stearate fumarate, Opadry) Patients who have abnormal ECG with clinical significance: congenital long QT syndrome or known family history of long QT syndrome; QTc > 450ms (male) or QTc > 470ms (female); patients who have ventricular or atrial tachyarrhythmia with clinical significance, etc Family planning participants (including male subjects) during or within three months after the trial Patients with a history of blood donation within 3 months before the trial Patients with a history of smoking (more than 5 cigarettes or products with equivalent nicotine per day), alcoholism (more than 14 units of alcohol per week: 1 alcohol unit equals 10 mL or 8 g pure alcohol; 25 mL 40% liquor, 330 mL 5% beer, 175 mL 12% red wine equals 1.0, 1.5, 2.0 alcohol units respectively), drug abuse and addiction history
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-110.0, SCAD Addison Disease Ankylosing Spondylitis Antiphospholipid Antibody Syndrome Celiac Disease Crohn Disease Dermatomyositis Polymyositis Guillain-Barre Syndrome Hepatitis, Autoimmune Graves Disease Hashimoto Thyroiditis Multiple Sclerosis Myasthenia Gravis Pernicious Anemia Polymyalgia Rheumatica Primary Biliary Cirrhosis Psoriasis Rheumatoid Arthritis Systemic Sclerosis Sjögren Syndrome Systemic Lupus Erythematosus Takayasu Arteritis Type 1 Diabetes Mellitus Ulcerative Colitis Uveitis Vasculitis Vitiligo Raynaud Adults age 18 to 110 Residence in Olmsted County. Note: if sufficient numbers cannot be reached using only Olmsted County, will expand to the 27-county region None
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Generalized Myasthenia Gravis Study participant must be ≥18 years of age, at the time of signing the informed consent Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participant's history and supported by previous evaluations Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at Screening (Visit 1).The presence of autoantibodies may be confirmed with repeat testing at Visit 1 Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa at Visit 1 Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 (with >3 points from non-ocular symptom) AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2) Study participant is considered for additional treatment such as intravenous immunoglobulin g (IVIg) or plasma exchange (PEX) by the Investigator Study participant has a known history of hyperprolinemia Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP) Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI) will be excluded Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Visit 1 or Visit 2 Study participant has a history of a solid organ transplant or hematopoietic stem cell/marrow transplant
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Patients with Myasthenia Gravis Age ≥18 years none
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 8.0-15.0, Reproductive Physiological Processes Pediatrics Adolescent Health Adolescent Developement Part 1-Pre-menarche monitoring (holding pattern) Age 8-14.5 years old Healthy weight, defined as having a body weight >85% of expected (EBW) and a body mass index (BMI) <99th percentile Some breast development Pre-menarche Taking or planning to take medications that affect reproductive hormones in the next 2 to 3 years (e.g. birth control pills) Planning to move more than 60 miles from the CRU within the next 2 years Chronic medical condition, including but not limited to diabetes mellitus, congenital adrenal hyperplasia, cystic fibrosis, sickle cell disease, inflammatory bowel disease, juvenile rheumatoid arthritis, and lupus First-degree relative with polycystic ovarian syndrome, premature ovarian insufficiency, hypogonadotropic hypogonadism, or other pubertal development disorder Excessive exercise (defined as running >20 miles per week or its equivalent) Pregnancy Part 2 Post-menarche cycle tracking Age at menarche 10-14.5 years old Healthy weight, defined as having a body weight >85% of expected (EBW) and a body mass index (BMI) <99th percentile Approximately less than or equal to 6 months post-menarchal (will typically have completed 4 or fewer menstrual cycles) Biochemical normal thyroid hormone, prolactin, and testosterone levels
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 16.0-999.0, Appendicitis Any patient presents with right iliac fossa pain Age 16 years old or more Previous appendicectomy
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 10.0-24.0, HIV HIV negative or unknown cohort Female to 20 years of age self-report HIV status as negative or unknown (no HIV testing within the past 6 months not pregnant, does not suspect pregnancy, and does not express a desire to become pregnant over the next 18 months is willing to sign a release for medical records (to obtain clinic data on service use) plans to reside in the current location for the next 18 months has not been part of the other planned formative research activities HIV positive cohort Female to 24 years of age pregnant
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 2.0-999.0, Upper Extremity Defects different traumatic defects in the hand and upper extremities who underwent reconstruction by random pedicle abdominal flaps Patients with incomplete data about the patient and the surgery
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Metastatic Breast Cancer Adult women with locoregionally recurrent or metastatic disease not amenable to curative therapy 2. Eastern Cooperative Oncology Group (ECOG) 0-2 3. Hormone receptor positive 4. No prior systemic anti-cancer therapy for advanced ER+ disease ( hormonal therapy) 5. Measurable disease defined by revised (version 1.1), or bone-only disease 6. normal laboratory values 7. Postmenopausal or premenopausal with oophorectomy (medical or surgical) Patients with advanced, symptomatic, visceral spread that are at risk of life threatening complication in the short term 2. Prior (neo) adjuvant treatment with same aromatase inhibitor type with DFI =< 12 months from completion of treatment. 3. Known uncontrolled or symptomatic central nervous system metastases 4. Second primary malignancy 5. Serious uncontrolled intercurrent infections or intercurrent medical or psychiatric illness 6. unable to swallow tablets, or malabsorption patients. 7. unwilling or unable to comply with study protocol or unable to meet the follow up. 8. patients who researchers considered were not suitable to participate
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-44.0, Fetal Alcohol Spectrum Disorders Drinking pregnant women who are defined as women who Drank in the 3 months prior to pregnancy and/or Drank alcohol at least once at any time during the prenatal period Gestational age less than or equal to 16 weeks, but not more than 20 weeks at intake Non-drinking pregnant women who are defined as women who have not had any alcohol during the past 30 days Gestational age >20 weeks
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 5.0-999.0, Acute Pharyngitis Pediatrics and adult > 5 years of age Patients complain of acute sore throat likely due to infective pharyngitis/tonsillitis as judged by the treating clinician Onset of symptoms within 7 days Patient or caregiver has the capacity and willingness to give consent and complete the trial paperwork, including the symptom diary Not on antibiotics for AP Pregnant or lactating mother Recent use of oral or inhaled steroids within 7 days Presence of an alternative diagnosis e.g. pneumonia, croup, bronchiolitis Known immune-deficiency (e.g. HIV, active chemotherapy or advanced cancer) Complicated acute sore throat that hospital admission is required (e.g. completely unable to swallow, very systemically unwell, or peritonsillar abscess) Presence of clear contraindication for steroids use (refer to the British National Formulary (BNF) list of contraindications) Requirement for the live vaccine in the next 7 days
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Adenocarcinoma Adenocystic Carcinoma Anal Cancer Appendix Cancer Brain Tumor Glioblastoma Astrocytoma Bile Duct Cancer Cholangiocarcinoma Bladder Cancer Bone Cancer Synovial Sarcoma Chondrosarcoma Liposarcoma Sarcoma, Kaposi Sarcoma,Soft Tissue Sarcoma Osteosarcoma CNS Cancer Brain Stem Neoplasms Breast Cancer Cervical Cancer Colorectal Cancer Rectal Cancer Colon Cancer Esophageal Cancer Esophagus Cancer Cancer of Colon Pancreatic Cancer Cancer of Pancreas Testis Cancer Testicular Cancer Ureter Cancer Renal Cell Carcinoma Kidney Cancer Gestational Trophoblastic Tumor Head and Neck Neoplasms Parotid Tumor Larynx Cancer Tongue Cancer Pharynx Cancer Salivary Gland Cancer Acute Myeloid Leukemia Chronic Myeloid Leukemia Acute Lymphoblastic Leukemia Multiple Myeloma Non Hodgkin Lymphoma Carcinoid Tumor Lung Cancer Neuroendocrine Tumors Mesothelioma Thyroid Cancer Parathyroid Neoplasms Adrenal Cancer Small Bowel Cancer Stomach Cancer Liver Cancer Hepatic Cancer Melanoma Skin Cancer Unknown Primary Tumors Uterine Cancer Fallopian Tube Cancer Ovarian Cancer Prostate Cancer Vaginal Cancer Penile Cancer Vulvar Cancer Waldenstrom Macroglobulinemia Cancer, Advanced Thymus Cancer Nasopharyngeal Carcinoma Multiple Endocrine Neoplasia Pheochromocytoma Small Cell Carcinoma Pulmonary Carcinoma Patient or representative provides written informed consent Patient is diagnosed with advanced malignancy Patient is willing to be treated for this malignancy according to a plan determine by them and their physician patient will be willing to have regular follow up visits as part of their standard of care patient is not a candidate or does not desire any treatment for their disease
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Muscular Dystrophies Duchenne muscular dystrophy Becker muscular dystrophy LGMD myotonic dystrophy type 1 and type 2 metabolic myopathy mitochondrial myopathy FSHD1 and 2 myasthenia history of cardiac surgery history of thoracic surgery brain traumatism
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 13.0-19.0, Polycystic Ovary Syndrome Puberty Disorders Ovulation Disorder Girls age 13 years, with clinical or biochemical HA (defined as hirsutism and/or elevated Tanner stage-specific free testosterone concentrations) that are 4-6 years post-menarche Screening labs within age-appropriate normal range Volunteers who are 18-19 y old must be willing and able to provide written informed consent When the subject is a minor (i.e., age < 18 y), the subject and custodial parents must be willing and able to provide written informed assent and consent, respectively Willingness to strictly avoid pregnancy (using non-hormonal methods) during the time of study Age < 13 or > 19 y Girls ≤ 3 years and ≥ 7 years post-menarche will be excluded Being a study of androgen excess in adolescent girls with HA, men and boys are excluded Inability to comprehend what will be done during the study or why it will be done Precocious puberty (breast development before age 7) Primary amenorrhea (no menses by age 16) BMI-for-age < 5th percentile Patients currently enrolled in another research protocol will be excluded, except for those enrolled in IRB-HSR 17633 DENND1A Obesity due to genetic syndrome (e.g. Prader-Willi syndrome) Cushing syndrome
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 10.0-99.0, Therapeutic Abortion With Complications Personality Disorders Trauma, Psychological Reproductive Behavior Post Traumatic Stress Disorder Depression, Reactive Pregnancy Prenatal diagnosis of fetal abnormality Adequate comprehension Legal to TToP procedure after psychiatric clinical evaluation Acceptance on a voluntary basis Incapacity Acute psychosis
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-999.0, Myasthenia Gravis Age >14. 2. Clinical Diagnosis of MG with supporting evidence: 1. unequivocal clinical response to pyridostigmine 2. positive antibody testing 3. decrement >10% in repetitive nerve stimulations study (RNS) . 3. Willingness to sample collection, imaging study and other disease-related examinations and assessments. 4. Patients with informed consent History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue. 2. Age ≤14 years. 3. Severe anxiety, depression or schizophrenia. 4. Cognitive impairment or mini-mental state examination (MMSE) score ≤24. 5. Severe systemic illness with life-expectancy less than 4 years. 6. Unwillingness to consent for collection of biological samples. 7. Inability to provide informed consent
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2
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.25-18.0, Duchenne Muscular Dystrophy Pompe Disease (Late-onset) months -18 years old boys and girls Serum transaminase levels (serum ALT and / or AST levels> 1.52 upper limit of normal (ULN)) for at least 3 months The willingness of the patient and / or legal representative to sign the written consent form Patients less than 3 months Patients with a known history of liver disease Patients with a known history of muscle disease Patients with a known history of rheumatologic disease Patients with clinical history or physical examination findings that support the possibility of liver disease (Jaundice, variceal bleeding, hepatomegaly, splenomegaly, ascites) ICU patients Patients with known congenital anomalies Patients with organ failure Patients with elevated serum GGT, Total Bliribun or Direct Bilirubin levels
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Cancer Pregnancy Cancer in pregnancy CT-treated arm Histological proven cancer during pregnancy (any type and stage) (Former) participation in part I.IA of the CIP-study S25470 (and I.IB for the placental sub study) Treatment during pregnancy with one or a combination of the following chemotherapeutic agents Cyclophosphamide Anthracyclines Taxanes Platinum derivates Gestational age (GA) at birth ≥24 weeks Cancer in pregnancy GA at birth <24 weeks (miscarriage or termination of pregnancy) (placental study) Mentally disabled women or patients who have a significantly altered mental status that would prohibit the understanding and giving of informed consent Any comorbidity that is associated with an enhanced risk of placental pathology or FGR such as hypertensive disorders, preeclampsia, (gestational) diabetes, SLE, Crohn's disease, renal or cardiac pathology (healthy pregnant controls)
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis, Generalized Patient must be at least 18 years of age Patient must have Generalized Myasthenia Gravis at the time of screening Concomitant immunosuppressive drugs must be deemed necessary by the investigator Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient Patient is pregnant or lactating
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Patients with Myasthenia Gravis Age ≥18 years a proper communication with the patient is not possible an informed consent could not be signed a patient reject a participation or requires breaking up
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Main 1. Diagnosis of Myasthenia Gravis (MG) supported by a positive serologic test for anti-AChR or anti-MuSK antibodies at screening. 2. Myasthenia Gravis Foundation of America (MGFA) clinical classification II to IV at screening. 3. Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of 6 or greater at screening, with at least 4 points attributed to nonocular items. 4. If receiving immunosuppressive drugs (ie, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, cyclophosphamide), therapy must be ongoing for at least 6 months, with stable dosing ongoing for at least 3 months before screening. Participants receiving azathioprine must be on a stable dose for at least 6 months before screening. 5. If receiving oral corticosteroids, therapy must be ongoing for at least 3 months, with a stable dose at least 1 month before screening. Corticosteroids, including dexamethasone, must be given as oral, daily or every-other-day therapy, as opposed to pulse therapy. 6. If receiving cholinesterase inhibitors, therapy with a stable dose is required at least 2 weeks before screening. 7. The doses of concomitant standard background therapy must be expected to remain stable throughout the study unless dose reduction is required due to toxicities. Allowed background therapy is defined as no more than a cholinesterase inhibitor ± corticosteroid ± 1 steroid-sparing immunosuppressive drug (limited to azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide). Participants must be on at least one allowed background medication. Main Presence of a thymoma (previous history of a fully encapsulated thymoma removed ≥ 12 months before screening is allowed) or history of invasive thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening. 2. History of thymectomy within 12 months before screening. 3. MGFA class I or V. 4. Received intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg), or plasmapheresis/plasma exchange within 4 weeks before screening, or an expectation that any therapy besides the participants standard background therapies may be used for treatment of MG (eg, a rescue therapy) between screening and dosing. 5. Chronic obstructive pulmonary disease (COPD) or asthma with a pre-bronchodilatory forced expiratory volume in 1 second (FEV1) <50% of predicted normal. Note: FEV1 testing is required for participants suspected of having COPD or asthma. 6. Received rituximab, belimumab, eculizumab, or any monoclonal antibody for immunomodulation within 6 months before first dosing. Participants with prior exposure to rituximab must have CD19 counts within the normal range at screening. 7. Known autoimmune disease other than MG that could interfere with the course and conduct of the study. 8. Received a live vaccine within 4 weeks before screening or has any live vaccination planned during the study. 9. Opportunistic infection ≤12 weeks before initial study dosing or currently receiving treatment for a chronic opportunistic infection, such as tuberculosis (TB), pneumocystis pneumonia, cytomegalovirus, herpes simplex virus, herpes zoster, or atypical mycobacteria. A mild, localized herpes simplex infection within 12 weeks of study dosing is allowed, as long as the lesion has resolved without systemic therapy prior to Day 1
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-49.0, Dysmenorrhea Women within reproductive age (15 to 49 years); 2. Participants who complain about dysmenorrhea with at least grade II level. 3. Self-reported lower abdominal and pelvic, medial anterior thigh pain or low back pain associated with the onset of menses and lasting in 72 hours. 4. Some accompanied symptoms as dizziness and syncope, cramping, nausea, vomiting, diarrhea, headaches, and fatigue present compatible with the menses cycle were also included Dysmenorrhea resulting from the use of an intra-uterine device (IUD); 2. Mild or infrequent dysmenorrhea. 3. Dysmenorrhea with possible underlying tumor or malignancy
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-75.0, Ocular Myasthenia Gravis Age >18 years and<75 years; 2. Clinical Diagnosis of MG with supporting evidence: Patients with ocular muscle type MG who have never received treatment meet one of the diagnoses (1) and (2)-(5): (1) The patient's symptoms involve the extraocular muscles, except for drooping eyelids and diplopia, no other clinical symptoms; (2) unequivocal clinical response to pyridostigmine; (3) positive acetylcholine receptor antibody or musk antibody; (4) decrement of more than 10% in repetitive nerve stimulations study (RNS); (5) the "trembling" of the single fiber electromyography (SFEMG) is broadened with or without blockade; 3. Willingness to sample collection, imaging study and other disease-related examinations and assessments; 4. The results of pregnancy tests for female subjects with fertility during the screening period should be negative and effective contraception was used by the patient and her spouse during the study period; 5. Patients with informed consent; 6. Predicted survival time is longer than 3 years History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue; 2. Patients who may have other diseases that may lead to eyelid drooping, peripheral muscle weakness or diplopia; 3. Age ≤18 years or ≥75 years; 4. Patients who have been taking glucocorticoids or associated immunosuppressants due to other immune system diseases; 5. Patients who cannot use immunosuppressants due to other chronic diseases; 6. Patients who are unable to cooperate with follow-up and self-assessment due to severe mental illness or cognitive impairment; 7. Pregnant women, lactating women and patients with fertility plans during the trial; 8. Patients who have suffered from severe infections or malignant tumors in the last 1 month and are unable to receive immunosuppressants treatments; 9. Patients who are not willing to cooperate with repeated frequency electrical stimulation tests and chest CT examinations; 10. Patients who are not willing to participate in this study; 11. Patients who are unable to sign informed consent; 12. Predicted survival time is shorter than 3 years; 13. Patients who are not suitable to participated in the trial after researchers' assessment
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Idiopathic Pulmonary Fibrosis Patients suspected of having an ILD who are having cryobiopsies performed BMI > 35 DLCO < 35% FVC < 45% Pulmonary hypertension Cardiac or other severe comorbidity that will increase the risk of complications
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Acute Pancreatitis Quality of Life Acute pancreatitis Must be able to response an online survey • N/A
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.5-59.0, Malaria Falciparum Malaria Vivax Malaria Above 6 months old to 59 years old Mono-infection with P. falciparum or P. vivax, with parasitemia of: P. falciparum: 1000-100 000 asexual forms per µl; P. vivax : ≥ 250 per µl Axillary temperature ≥37.5 °C or oral/rectal temperature of ≥38 °C Ability to swallow medication Ability and willingness to comply with the study protocol for the duration of the study and to comply with the study visit schedule Informed consent from the patient or from a parent or legal guardian in the case of children less than 18 years old Informed assent from any minor participant aged 12 years; and Consent for pregnancy testing from female of child-bearing potential and from their parent or guardian if under 18 years old Severe malnutrition Mixed Plasmodium species detected by microscopy Presence of severe malnutrition (defined as a child whose weight-for-height is below -3 standard deviation or less than 70% of the median of the NCHS/WHO normalized reference values, or who has symmetrical oedema involving at least the feet or who has a Mid Upper Arm Circumference [MUAC] <110 mm) Presence of febrile conditions due to diseases other than malaria (measles, acute lower tract respiratory infection, severe diarrhea with dehydration, etc.), or other known underlying chronic or severe diseases (e.g. cardiac, renal, hepatic diseases, HIV/AIDS) Regular medication, which may interfere with antimalarial pharmacokinetics History of hypersensitivity reactions or contraindications to any of the drug(s) being tested or used as alternative treatment Positive pregnancy test or breastfeeding; and Unable to or unwilling to take pregnancy test or to use contraception for women or child-bearing age and who are sexually active
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Myasthenia Gravis Myasthenia Gravis, Generalized Signed informed consent, demonstrating that the patient understands the procedures required for the study and the purpose of the study a. May be signed by parent or legal guardian in the case of minor or adult unable to provide consent 2. Male or female of all ages. 3. Diagnosis of Myasthenia Gravis by a physician Refusal to sign informed consent form 2. History of bariatric surgery, total colectomy with ileorectal anastomosis or proctocolectomy. 3. Postoperative stoma, ostomy, or ileoanal pouch 4. Participation in any experimental drug protocol within the past 12 weeks 5. Treatment with total parenteral nutrition
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2
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis diagnosed with myasthenia gravis and who are currently taking pyridostigmine and experienced pyridostigmine-related GI side effects within the past 7 days. 2. GSRS rating of at least Moderate discomfort on questions 5, 11, and 12. 3. Subjects must be willing and able to complete a GI symptom diary within a consistent timeframe on a daily basis. 4. Subjects must be able to tolerate a pyridostigmine dose of 30mg TID. 5. Must be clinically stable in judgement of treating neurologists for past 3 months. 6. Must have AchR antibody positive MG. 7. Subjects must be able to swallow liquids. 8. Subjects must be in good health as determined by their medical history, physical examination, vital signs, and laboratory tests. A subject with a medical abnormality may be included only if the investigator or designee considers that the abnormality will not introduce significant additional risk to the subject's health or interfere with study objectives. 9. Subjects must have signed an informed consent form indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study and comply with the study procedures and restrictions Key The for of a subject from enrollment in the study are as follows: 1. Any acute or chronic diseases which are associated with GI distress (such as nausea, vomiting, or diarrhea), which could interfere with the subjects' safety during the trial, expose them to undue risk, or interfere with the study objectives. 2. History or presence of hepatic, or renal disease or other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs. 3. History of substance abuse, known drug addiction, or positive test for drugs of abuse or alcohol. 4. Patients currently using marijuana for any reason (medical or recreational). 5. Known hypersensitivity to pyridostigmine, or to ondansetron or similar 5-HT3 serotonin receptor antagonists. 6. ECG changes including QT interval prolongation and congenital long QT syndrome. Electrolyte abnormalities (e.g., hypokalemia or hypomagnesemia), congestive heart failure, bradyarrhythmia's or other medicinal products that lead to QT prolongation. 7. Treatment with drugs affecting peripheral cholinergic transmission within 1 month of study entry (with the exception of pyridostigmine). 8. Subjects unlikely to co-operate during the study, and/or be questionably compliant in the opinion of the investigator. 9. Patients currently being treated with narcotics. 10. Patients being treated with aminoglycoside antibiotics, which are contraindicated in myasthenia gravis. 11. Patients unable to be contacted in case of an emergency. 12. Intake of an investigational drug within 30 days of study entry. 13. Pregnancy and women of childbearing potential not willing to follow the birth control requirements as described in the informed consent or breastfeeding. 14. History or presence of obstructive pulmonary disease or urinary obstruction (contraindication for pyridostigmine). 15. This use of selective serotonin reuptake inhibitors (SSRI's)
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 13.0-60.0, Psoriasis minimum age 13 years maximum age 60 years both males and females affected with mild, moderate and severe psoriasis hypertension cardiovascular disorders pregnancy lactation renal failure liver failure hypersensitivity to drug
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Spinal Muscular Atrophy SMA All patients with a confirmed SMA diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results There are no for the registry
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-22.0, Acute Myeloid Leukemia All patients must be enrolled on APEC14B1 and consented to Screening (Part A) prior to enrollment and treatment on AAML1831. Submission of diagnostic specimens must be done according to the Manual of Procedures). Risk stratification will not be possible without the submission of viable samples. Given there are multiple required samples, bone marrow acquisition techniques such as frequent repositioning or performing bilateral bone marrow testing should be considered to avoid insufficient material for required studies. Consider a repeat marrow prior to starting treatment if there is insufficient diagnostic material for the required studies Patients must be less than 22 years of age at the time of study enrollment Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease Patient must have 1 of the following >= 20% bone marrow blasts (obtained within 14 days prior to enrollment) In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy < 20% bone marrow blasts with one or more of the genetic abnormalities (sample obtained within 14 days prior to enrollment) A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell [WBC] count >= 10,000/uL with >= 10% blasts or a WBC count of >= 5,000/uL with >= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment) ARM C: Patient must be >= 2 years of age at the time of Late Callback ARM C: Patient must have FLT3/ITD allelic ratio > 0.1 as reported by Molecular Oncology Patients with myeloid neoplasms with germline predisposition are not eligible Fanconi anemia Shwachman Diamond syndrome Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21 Any other known bone marrow failure syndrome Any concurrent malignancy Juvenile myelomonocytic leukemia (JMML) Philadelphia chromosome positive AML Mixed phenotype acute leukemia Acute promyelocytic leukemia
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 6.0-35.0, Amblyopia Anisometropia Patients with anisometropic amblyopia who had proper optical correction using spectacles or contact lenses for the past 6 weeks. Anisometropic amblyopia will be defined as difference > 0.3 logMAR lines in best-corrected visual acuity with difference in the spherical equivalent of both eyes > 1 D, in the absence of other causes for amblyopia such as organic causes, or manifest strabismus < 5 PD by simultaneous prism and cover test. Patients will be included in the study only if they had no treatment for amblyopia for the past month other than spectacles and/or contact lenses Patients with development delay Prior ocular surgery Co-existing ocular disease Patients with allergy to patch material History of problems with prior use of virtual reality systems
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Pacemaker Lead Dysfunction All consecutive patients referred for lead extraction with class I and IIa indications according to 2017 HRS expert consensus statement on CIED lead management and extraction Patients primarily requiring surgical extraction
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Pelvic Organ Prolapse Stage II-IV pelvic organ prolapse Bothersome bulge symptoms At least 725 MET-minutes/week on International Physical Activity Questionnaire Short Form English-speaking Undergoing treatment of prolapse Surgery occurring at least 7 days from date of randomization (to allow for collection of at least 7 days of preoperative accelerometer data) Able and willing to follow up at 3 months for in-office exam Enrollment in another research study of pelvic organ prolapse Concomitant non-urogynecologic surgery Planned further surgery in the next 3 months or anticipated treatment which would result in prolonged inactivity (such as a cancer diagnosis) 3 months postoperatively
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-99.0, Myasthenia Gravis Signed Informed Consent Form. 2. Male or female ≥ 18 years of age. 3. A female participant is eligible to participate if she is of: 1. Non-childbearing potential (physically infertile, including women who have been menopausal for 2 years or more); 2. Childbearing potential, negative serum pregnancy test results at screening visits, and agree to consistently use acceptable and effective contraceptive methods until 14 days after the final visit. 4. A male participant must take effective contraception during this clinical trial or their heterosexual partner must take effective contraception. 5. Positive serologic test for AChR-Ab or MUSK-Ab at the screening visit and at least 1 of the following: 1. History of abnormal neuromuscular transmission test demonstrated by sign-fiber-electromyography or repetitive nerve stimulation OR 2. History of positive edrophonium chloride test OR 3. Participant has demonstrated improvement in MG signs on oral cholinesterase inhibitors as assessed by the treating physician Use of rituximab, belimumab, eculizumab or any monoclonal antibody/Fc-fusion biologic for immunomodulation within 6 months prior to screening. 2. Immunoglobulins given by SC, IV(IVIG), or intramuscular route, or plasmapheresis/plasma exchange (PE) within 4 weeks before screening. 3. Thymectomy performed < 12 months prior to screening. 4. Total IgG level <6g/L (at screening). 5. Participant has any laboratory abnormality (at screening) that, in the opinion of the investigator, is clinically significant, has not resolved at baseline, and could jeopardize or would compromise the participant's ability to participate in this study. 6. Have known autoimmune disease other than MG that would interfere with the course and conduct of the study (such as uncontrolled thyroid disease). 7. Have an active infection, a recent serious infection (i.e., requiring injectable antimicrobial therapy or hospitalization) within the 8 weeks prior to screening. 8. History of or known infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), or Mycobacterium tuberculosis. Participants must have negative test results for HBV surface antigen, HBV core antibody, HCV antibody, HIV 1 and 2 antibodies and a negative TB test at screening
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 13.0-35.0, Cornelia de Lange Syndrome Ages 13 to 35 years A diagnosis of CdLS as determined by a physician during routine care meeting the major and minor from CdLS guidelines Threshold for the presence of RB/SIB as reported on initial screening Children's Yale-Brown Obsessive Compulsive Scale Modified for Pervasive Developmental Disorders (CYBOCS-PDD) > 6 OR Aberrant Behavior Checklist (ABC) stereotypy subscale > 7) Being able to attend 4 visits over the course of 18 weeks at the Johns Hopkins Hospital No acute safety concerns or need for hospitalization due to psychotic, manic or depressive episode Not currently pregnant or lactating/breastfeeding. Whether a participant is pregnant or not will be determined by the participant/caregiver report based on date last menses. If there is any suspicion of pregnancy, the PI will confer with the family to obtain testing through the primary care provider Allergy to NAC Allergy to Quinine Contraindication to NAC (organ transplant; untreated or symptomatic gastric condition) Need for another medication with which NAC is contraindicated (antibiotics)
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-60.0, Myasthenia Gravis, Generalized fluctuating muscle weakness and fatigability classified as the Myasthenia Gravis Foundation of America (MGFA) clinical classification Type IIa-IVa more than 10% amplitude decrement in low frequency repetitive nerve stimulation,less than 10% amplitude increment in high frequency repetitive nerve stimulation anti-AChR antibody positivity understanding and assigning the informed consent form, and having a good compliance with treatment. meet all of the in above as well as at least one of the below refractory: insufficient response to full dose and course steroids treatment (0.75-1mg per kg per day for at least 3-6 months), failure to respond adequately to tacrolimus (no less than 3mg per day) for 3 months severe or intolerable adverse effects from conventional immunosuppressive therapy a tumor history except for thymoma a history of Type B and Type C hepatitis a history of tuberculosis or T-SPOT.TB tset positivity hepatic, renal and cardial insufficiency (baseline: ALT/AST>50U/L;BNP>200pg/ml） severe allergy or infection, or chronic or recurrent infection pregnancy hyperlipidemia participating other clinical trials
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 3.0-999.0, LGMD2E Gene based diagnosis of LGMD 2E Subscription of informed consent when applicable Lacking gene based diagnosis of LGMD 2E Lacking subscription of informed consent when applicable
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Diagnosis of MG with anti-AChR or anti-MuSK antibody. 2. MGFA Clinical Classification Class II, III, or IV. 3. MG-ADL score of 6 or greater at screening and at randomization with > 50% of this score attributed to non-ocular items. 4. QMG score of 11 or greater. 5. Subjects must be on: 1. Corticosteroids only, with no dose increase within 4 weeks prior to randomization, or 2. One allowed non-steroidal IST, with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization, or 3. Combination of (1) corticosteroids with no dose increase within 4 weeks prior to randomization and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization. Allowed ISTs, alone or in combination with corticosteroids, are azathioprine, mycophenolate mofetil, and mycophenolic acid Receipt within the 4 weeks prior to Day 1: 1. Cyclosporine (except eye drops) 2. Tacrolimus (except topical) 3. Methotrexate 2. Current use of: 1. Prednisone > 40 mg/day or > 80 mg over a 2-day period (or equivalent dose of other corticosteroids)
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-90.0, Myasthenia Gravis fluctuating muscle weakness and fatigability, along with one of the below more than 10% amplitude decrement in low frequency repetitive nerve stimulation,less than 10% amplitude increment in high frequency repetitive nerve stimulation anti-AChR or MuSK antibody positivity positive to the neostigmine test understanding and assigning the informed consent form, and having a good compliance with the follow up excluding the possible diagnosis of Lambert-Eaton syndrome, congenital myasthenia syndrome,botulism injection, chronic progressive extraocular ophthalmoplegia, etc participating other clinical trials poor compliance to the follow up
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2
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-18.0, Healthy Pre and post-pubertal females 2. Ages 0-18 years old 3. Tanner Stage I V 4. Undergoing routine outpatient surgical procedure 1. Hernia repair (inguinal, umbilical, epigastric) 2. Excision of benign mass 3. laparoscopic cholecystectomy Previous diagnosis of an illness associated with premature ovarian failure (Turner's syndrome, Fragile X permutation carrier) or endocrine disorder associated with irregular menstrual cycles (Cushing's disease, poorly-controlled Thyroid disease, hyperprolactinemia, polycystic ovary syndrome, and congenital adrenal hyperplasia) or insulin-dependent diabetes mellitus or autoimmune disorders 2. Previous diagnosis of any malignancy or any history of systemic/local chemotherapy, radiation therapy, or stem-cell transplant. 3. Previous surgical excision of one ovary or both ovaries 4. Pregnant females 5. All inpatient surgical patients 6. Undergoing non-routine outpatient surgical procedures 1. Central venous catheter placement 2. Supprelin insertion/removal 3. Breast mass excision 4. Gastrostomy tube insertion
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-0.058, Maternal Depressive Disorder Complicating Childbirth Preterm Birth Maternal Anxiety Disorder Complicating Childbirth Mother Commitment to finish all questionnaires and to participate in the creation of a CD to be played to her baby Older than 18 years of age Infant • Gestational age between 26 0/7 and 30 6/7 weeks Mother • Younger than 18 years Infant • Major congenital or chromosomal anomalies
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-80.0, Anti-MAG Neuropathy Patients with a confirmed diagnosis of monoclonal IgM associated with MGUS with anti-MAG activity (titer of > 10'000 BTU) and demyelinating neuropathy defined by electrophysiological according to EFNS/PNS PDN guideline, 2010 Clear clinical signs of disability Adequate hepatic and renal function Patients with total serum IgM levels >30 g Hematological malignancy, prior malignancy of any organ system (except BCC) Prior immunosuppression: No IVIG in previous 3 months, no previous cyclophosphamide or biologicals in prior 6 months Other neurological, neuromuscular, rheumatologic or orthopedic condition with significant impact on the capabilities of walk preventing evaluation of neurological scores
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 14.0-999.0, Cerebral Vein Thrombosis patients aged 14 years or older and weighing more than 50 kg Recent diagnosis of symptomatic cerebral venous thrombosis confirmed by MRI modalities, CT venography, or conventional angiography Any absolute contraindication to anticoagulation Impaired renal function (CrCl < 30 mL/min using Cockcroft-Gault equation) Pregnancy or lactation at randomization mandatory other blood thinners use (e.g. Aspirin)
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Must have a documented diagnosis of Myasthenia Gravis 2. Must have ocular (eye drooping) and/or bulbar (speech) symptoms 3. Must be over the age of 18 4. Must reside in the US for the duration of the study 5. Must be able to read, understand, and write in English 6. Must have a smartphone supported by the doc.ai research app (iOS and Android) None
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Hydrocephalus, Normal Pressure Diagnosis of idiopathic normal pressure hydrocephalus and planned treatment with shunt surgery Ongoing anticoagulation treatment Ongoing Clopidogrel treatment Mini mental state examination results of 15 or lower Contraindications to magnetic resonance imaging
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Coronary Heart Disease the presence of an established diagnosis in patients with coronary artery disease unstable angina pectoris (NS). When making a diagnosis, the recommendations of ESH / ESC (2015) and RCO / WHO (2014) were used stable exertional angina (SSN) I-III FC. When making the diagnosis, the IHD classification was used, adopted at the IV Congress of Cardiologists of the Republic of Uzbekistan (2000), as well as in accordance with the recommendations of ESH / ESC (2019) and RCO / WHO (2017) The patient's refusal to participate in the study Pregnancy and lactation Severe and unstable condition of the patient, making it difficult to conduct a questionnaire (for ethical reasons) Acute violation of cerebral circulation History of acute or chronic psychosis The presence of concomitant acute diseases or chronic diseases in the acute stage. Throughout the study, all patients were assigned the right to voluntarily withdraw from the study at their own request, notifying the researcher in writing or orally
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Schizophrenia Major Depressive Disorder A total of 300 subjects will be included in the trial Adults (18 years old and above) Male and Female N/A
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Postpartum Hemorrhage Women with major postpartum hemorrhage (1.0 liter or more) Language difficulties requiring an interpreter or translator
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Ocular Myasthenia Gravis Patients with OMG diagnosis and its generalization who consulted between January 1990 to January 2017 at Fondation Hospital Adolphe de Rothschild and registered in the database OMG differential diagnoses
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2
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-80.0, Myasthenia Gravis Myasthenia Gravis Crisis Meeting the diagnosis of myasthenia gravis Malignant tumor or other serious chronic disease
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2
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-999.0, Systemic Autoimmune Diseases In order to be eligible to participate in this study, an individual must meet all of the following as per respective group: 1. Stated willingness to comply with all study procedures and availability for the duration of the study 2. Male or female age greater than or equal to 15 years old with no upper age limit. 3. Ability of subject to understand and the willingness to sign a written informed consent and/or assent document. 4. For Healthy Volunteers Group Age greater than or equal to 15 with no upper age limit No history of autoimmune diseases and in good general health as evidenced by medical history. 5. For symptomatic COVID-19 group must have laboratory evidence (positive PCR for or other test developed after this proposal gets approved and is available through the Clinical Center) and one of the signs and symptoms associated with COVID-19 infection (e.g. fever or chills, cough, shortness of breath or difficulty breathing, fatigue, muscle or body aches, headache, new loss of taste or smell, sore throat, congestion or runny nose, nausea or vomiting, diarrhea). 6. For post COVID-19 study visits must have laboratory evidence (e.g. positive PCR for SARS COV-2, antibody against SARS-COV-2 or other test developed after this proposal gets approved) of current or prior exposure to COVID-19. Alternatively, patients should have documented evidence of having received one of the SARS-COV-2 vaccines that may become available during the study. 7. For subjects with known COVID exposure, they must fulfill one of the following A minimum of 28 days has passed since the initial positive test date AND B. Resolution of fever was greater than or equal to7 days ago (without the use of fever-reducing medications) AND C. Resolution of respiratory symptoms was greater than or equal to7 days ago, OR A minimum of 10 days has passed since the initial positive test AND B. A minimum of 2 consecutive oropharyngeal swabs OR 2 consecutive nasopharyngeal swabs or 2 saliva testing collected greater than or equal to 24 hours apart that are negative for SARS CoV-2 PCR A positive PCR requires restarting the series of swabs (wait 5 days to retest) 8. Specific for Systemic Autoimmunity Diseases Group Age greater than or equal to 15 years with no upper age limit Systemic lupus erythematosus (SLE): Meets at least 4 of 11 modified American College of Rheumatology (ACR) (1997) Revised for the Classification of Systemic Lupus Erythematosus or Systemic Lupus International Collaborating Clinics (SLICC) classification for systemic lupus erythematosus Anti-neutrophil cytoplasmic antibody associated vasculitis (AAV): Meet Revised 1990 ACR for Granulomatosis with polyangiitis (GPA) or the 2012 Chapel Hill Nomenclature for microscopic polyangiitis (MPA) Idiopathic inflammatory myopathies (IIM):Meets the 2017 EULAR/ACR Classification for Adult and Juvenile idiopathic inflammatory myopathies and their major subgroups An individual who meets any of the following will be excluded from participation in this study: 1. Pregnant and lactating subjects will be excluded because pregnancy and lactation modify immune responses and may interfere with correlations. 2. Concomitant medical problems which would confound the interpretation of studies gathered by this protocol. Included in this is the presence of HIV in the blood, active malignancies, or other significant medical conditions that may interferes with interpretation of studies. 3. Concomitant medical, surgical or other conditions for which inadequate facilities are available to support their care at NIH. 4. Inability or unwillingness to comply with follow up requirements (e.g. distance, social, physical limitations). 5. Any comorbidity of medical or psychological/psychiatric condition or treatment, that in the opinion of the Principal Investigator, would the subjects from the research studies (e.g. Patient requiring urgent and/or acute medical care, surgical or other procedures) 6. Unwilling to participate in research studies or to provide research samples or data 7 for the (optional) vascular studies: -Healthy volunteers with known history of coronary artery disease, peripheral vascular disease or atherosclerosis. 8 for the (optional) scan: -Individuals younger than 18 years old will be excluded given the radiation exposure 9. Subjects with SAD and Healthy volunteers younger than 18 years old will be excluded from chest x-rays unless clinically indicated
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 1.0-99.0, Myasthenia Gravis Child or adult patients, living or deceased, presenting or having presented a myasthenic syndrome and a COVID-19 infection 2. Myasthenic syndrome is established by Either the presence of a specific antibody Either the presence of specific electrophysiological abnormalities Either an evocative symptomatology improved by a therapeutic test with an acetylcholinesterase inhibitor Either one or two pathogenic mutation (s) in a gene involved in congenital myasthenic syndromes (dominant or recessive disease). 3. COVID-19 infection is established by Either a positive PCR test Or a specific chest scanner Either a positive serology Either a clinical syndrome of COVID-19, validated by a committee of experts. 4. Patients affiliated or beneficiaries of a social security scheme 5. For living patients: patients who have been informed of the study and have not exercised their right of opposition or parents or holders of parental authority who have been informed of the study and have not exercised their right opposition. For deceased patients: beneficiaries or parents / holders of parental authority having been informed of the study and not having exercised their right of objection Persons placed under judicial protection
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 14.0-999.0, Adolescent Pregnancy All subjects must be able to read and write Spanish RCT will be limited to pregnant adolescents who are between the ages of 14-19 years and 12-34 weeks gestational age. Other caregivers (age 14 years or older) will be eligible if they are planning to be involved in infant care Only pregnant adolescents receiving outpatient prenatal care at INMP or San Bartolomé will be approached as potential participants Only pregnant adolescents who have been cleared by their attending doctor/nurse to engage in the approach/recruitment and interview procedure will be approached as potential participants All infants born to adolescents enrolled during pregnancy will be eligible to be enrolled after birth Anyone unable to read and write Spanish Any pregnant adolescent known to have a severe fetal anomaly will not be approached Infants who are advised not to participate by their attending doctor or pediatrician Children or adolescents who are wards of the state will not be enrolled
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-41.0, Habitual Abortion Recurrent Pregnancy Loss Fertility Disorders Miscarriage Women with ≥ 2 consecutive pregnancy losses (miscarriages or biochemical pregnancies) ≤ completed gestational week 10 after ART with the present partner or with an egg/semen donor BMI ≥35 2. Age ≥41 3. Significant uterine malformation(s) 4. Known parental balanced chromosomal translocations 5. ≥2 previous pregnancies with fetuses with known abnormal karyotype 6. Patients with IgA deficiency, IgA-autoantibodies or hyperprolinaemia 7. Treatment with medication interacting with prednisolone CYP3A4-inhibitors (fx erythromycin, itraconazole, ritonavir, lopinavir), CYP3A4-inductors (fx phenobarbital, phenytoin og rifampicin), loop diuretics, thiazides, amphotericin B, beta2-agonists, antidiabetics, interleukin-2, somatotropins, anticholinergics and regular treatment with NSAIDs. 8. Patients with moderate/severe hypertension, diabetes mellitus, heart insufficiency, severe mental disorders, Cushing syndrome, myasthenia gravis, ocular herpes simplex, pheochromocytoma, systemic sclerosis, and moderate/severe renal dysfunction. 9. Patients with a clinical or biochemical profile indicating need for heparin or levothyroxine treatment during pregnancy 10. Previous treatment with IVIg 11. Allergy to prednisolone and/or IVIg 12. AMH <4 pmol/L. If transfer of donor egg is planned for her IVF cycle, the AMH value will not be an criterion
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 9.0-26.0, Papillomavirus Infections Boys and Girls 9 to 15 Years Must not have had coitarche and does not plan on becoming sexually active during the vaccination period Women 16 to 26 Years Has never had a Papanicolaou (Pap) test or only had normal Pap test results A lifetime history of 0 to 4 male and/or female sexual partners Cohort 0 Participants Received 1 dose of 9vHPV vaccine at least 1 year prior to enrollment and did not receive a second dose of any HPV vaccine All Participants Known allergy to any vaccine component History of severe allergic reaction that required medical intervention Thrombocytopenia or any coagulation disorder Females only: participant is pregnant or expecting to donate eggs during day 1 through month 7 Currently immunocompromised, or been diagnosed with immunodeficiency Had a splenectomy Receiving or has received immunosuppressive therapies within the last year Received any immunoglobulin product or blood-derived product within 3 months Has received more than 1 dose of an HPV vaccine (Cohort 0)
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 0.0-999.0, Herpes Simplex Virus Infection Five-months-old infants or younger Six-months-old infants or older
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Auto-immune Myasthenia Patient with generalized autoimmune myasthenia with Acetylcholine Receptor Antibodies (AChR Ab) or with antibodies to the muscle-specific receptor tyrosine kinase (MuSK Ab) Patient agreeing to participate in the therapeutic education program Signing consent Subject affiliated with a health insurance social protection regiment Patients without serological confirmation of myasthenia or pure ocular form Patient with cognitive impairment or behavioral problems that, in the investigator's opinion, will compromise their ability to comply with study procedures Refusal of the patient to participate in the study Patient who has already benefited from the therapeutic education program in myasthenia Age < 18 y.o Subject under safeguarding justice Subject under guardianship or under curatorship
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-100.0, Myasthenia Gravis Patients over age 18 2. Patients with a confirmed diagnosis of myasthenia gravis based on clinical including fatiguable weakness and supported by either serological (acetylcholine receptor, muscle specific kinase or anti-low-density lipoprotein receptor related protein 4 antibodies or electrophysiological testing (repetitive nerve stimulation of single fiber electromyography) 3. Myasthenia Gravis Federation of America class II-IV 4. Moderate to severe myasthenia gravis as defined by a quantitative myasthenia gravis score >10 or generalized myasthenia gravis impairment index score > 11 5. Patient able to give consent and is able and willing to complete all study procedures and activities Patients who are pregnant or breastfeeding 2. Patients not able to complete the study procedures or with an alternate diagnosis 3. Patients with recent thymectomy in the past 6 months 4. Patients receiving another biologic agent such as rituximab, belimubab, cyclophosphamide and eculizumab in the past 6 months prior to study entry 5. No IVIG or subcutaneous immunoglobulin within the past month 6. Patients on prednisone who have had alterations in prednisone dose over the past month prior to study entry 7. Patients will previous known allergy or severe adverse reaction to intravenous or subcutaneous immunoglobulin 8. Evidence of renal insufficiency (Cr>1.5 x elevated) or liver disease (transaminases > 2.5 x elevation) at screening
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Generalized Myasthenia Gravis Bullet list of each criterium: 1. Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 2. At least 18 years of age at the time of signing the informed consent form. 3. Diagnosed with generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by at least 1 of the following: 1. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation 2. History of positive edrophonium chloride test 3. Demonstrated improvement in Myasthenia Gravis (MG) signs upon treatment with oral acetylcholinesterase (AChE) inhibitors as assessed by the treating physician 4. Meeting the clinical as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa, or IVb Bullet list of each criterium: 1. Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of Investigational Medicinal Product. 2. Has any of the following medical conditions: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening 2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk. 3. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time adequately treated basal cell or squamous cell skin cancer carcinoma in situ of the cervix carcinoma in situ of the breast incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a or T1b). 4. Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Myasthenia Gravis Age ≥18 Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence: 1. positive clinical response to acetylcholinesterase inhibitor 2. positive AchR-Ab or MuSK-Ab testing 3. decrement >10% in repetitive nerve stimulations study (RNS) or increased jitter on single-fibre electromyography (SFEMG) MGFA clinical classification: I Baseline MG-ADL ≥ 3 Disease course from onset to enrollment ≤ 12 months Cooperation to followup Written informed consent Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment Thymectomy within 3 months prior to Screening Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue Consciousness, dementia or schizophrenia Pregnancy or lactation, unwillingness to avoid pregnancy Uncontrolled hypertension or diabetes, Liver or kidney dysfunction, Cataract, Severe osteoporosis, Femoral head necrosis; Hyperkalemia, HIV, Acute or chronic infection Other conditions that would preclude participation
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 18.0-999.0, Neurogenic Hypertension Hypertension Surgery Age ≥ 18 years old; 2. Preoperative MRI showed RVLM compression; 3. Primary hemifacial spasm without other secondary factors; 4. The office systolic blood pressure is higher than 140mmHg and the systolic blood pressure of ambulatory blood pressure is higher than 130mmHg; 5. Sign informed consent Secondary hypertension, such as renal hypertension, vascular hypertension, etc; 2. No antihypertensive drugs were taken after admission and ambulatory blood pressure monitoring showed that the blood pressure was within the normal range; 3. Pregnancy plan or lactation period during pregnancy or within 2 years; 4. Patients with congestive heart failure (NYHA grade II-IV), myocardial infarction and unstable angina pectoris within 6 months before enrollment; patients with severe heart diseases, such as cardiogenic shock, arrhythmia requiring treatment, heart valve disease, etc; 5. Severe cerebrovascular diseases (hypertensive encephalopathy, cerebrovascular injury, stroke, transient ischemic attack, etc.) within 6 months before admission; 6. Patients with severe liver disease or dysfunction (ALT or AST > 2 times the upper limit of normal reference value); 7. Patients with renal impairment (male serum creatinine > 2.0mg/dl (176 μ mol / L), female serum creatinine > 1.8mg/dl (159 μ mol / L)); 8. Other surgical contraindications, such as coagulation disorders, severe anemia, severe pulmonary infection, persistent state of asthma, upper respiratory tract infection, uncorrected respiratory failure, diabetic ketoacidosis, hepatic encephalopathy, hepatic coma, acute cerebral infarction, etc; 9. Type I diabetic patients; diabetic patients with poor glycemic control (FBG > 11.1 mmol / L) or type 2 diabetic patients with microalbuminuria; 10. Had Cushing's syndrome, coarctation of aorta, hyperthyroidism and other diseases in the past; 11. Patients can not cooperate with the test and follow-up, such as mental disorders, dementia, advanced tumor, etc; 12. If the antihypertensive drugs were adjusted within 2 weeks before admission, the patients were excluded or re enrolled after the blood pressure was stable for 2 weeks; 13. Preoperative anesthesia score ≥ grade III; 14. To screen those who participated in other clinical studies and used research drugs within 3 months before the trial, or planned to participate in other clinical studies during the trial period
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 15.0-19.0, Human Immunodeficiency Virus Female Aged 15-19 years HIV-negative (willing to self-test onsite in the presence of the Research Assistant who is also a trained HIV counselor) Has at least one current sexual partner she expects to meet at least twice in the next 3 months (not necessarily to have sex); this will provide opportunity for the participant to suggest/offer testing Resides in Siaya County and does not intend to relocate within 6 months from enrollment, to allow follow up to be completed and to conduct IDI with those selected Willing to be randomized into the partner self-testing or HTS referral groups Reporting no incident of sexual, physical, emotional, or economic abuse in the last 12 months from the partner she intends to offer (Appendix 4) or refer for testing (Appendix 3) in the next 3 months and does not believe the partner will abuse her if she offers the self-test kits or HTS referral coupon Has a male partner who has not tested for 6 months or whose HIV status is unknown to her Agrees to give consent to participate in the study Male HIV-positive Not willing to self-test onsite under in the presence of the Research Assistant Aged <15 years or >19 years Does not have a current sexual partner or does not expect to meet the partner at least twice in the next 3 months Does not reside in Siaya County or intends to relocate within the next 6 months Not interested in obtaining HIV self-tests or HTS referral coupon for purposes of suggesting testing to their sexual partners Reporting an incidence of sexual, physical, emotional or economic abuse in the last 12 months from the partner she would have offered self-test kit or referred for testing, or believes the partner may subject her to sexual, physical, emotional, or economic abuse if she offered them self-test kit or HTS coupon Has a male partner who tested within the last 6 months Does not give consent to participate in the study
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0
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 2.0-18.0, Generalized Myasthenia Gravis Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits). 2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent. 3. Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation 4. Meeting the clinical as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa. 5. Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant Generalized Myasthenia Gravis (gMG) therapy of adequate duration before screening. 6. Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (<15kg) historical values can be used). 7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. 1. Male participants: Male participants must agree to not donate sperm from the time the informed consent form was signed until the end of the trial. 2. Female participants: Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered Participants with Myasthenia Gravis Foundation of America (MGFA) class I, IVb, and V. 2. Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of investigational medicinal product (IMP). 3. Has any of the following medical conditions: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening. 2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk. 3. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the investigational medicinal product (IMP). Participants with the following cancers can be included at any time Adequately treated basal cell or squamous cell skin cancer Carcinoma in situ of the cervix Carcinoma in situ of the breast Incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a or T1b) 4. Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk 4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc). 5. A documented lack of clinical response to plasma exchange (PLEX). 6. Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary. 7. Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period. 8. The following results from these diagnostic assessments will be considered exclusionary: 1. Positive serum test at screening for an active viral infection with any of the following conditions Hepatitis B virus (HBV) that is indicative of an acute or chronic infection Hepatitis C virus (HCV) based on HCV antibody assay Human immunodeficiency virus (HIV) associated with a CD4 count <200 cells/mm3 with an acquired immunodeficiency syndrome (AIDS)-defining condition, such as: Cytomegalovirus retinitis with loss of vision, Pneumocystis jiroveci pneumonia, chronic intestinal cryptosporidiosis, HIV-related encephalopathy, Mycobacterium tuberculosis (pulmonary or extrapulmonary), or invasive cervical cancer 2. Positive nasopharyngeal swab polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). 9. Using the following prior or concomitant therapies: 1. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the investigational medicinal product (IMP). 2. Use of any monoclonal antibody within the 6 months before the first dose of the investigational medicinal product (IMP). 3. Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or Plasma exchange (PLEX) within 4 weeks before screening. 10. Total immunoglobulin G (IgG) levels <6 g/L at screening. 11. A known hypersensitivity reaction to efgartigimod or any of its excipients
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1
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The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy
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eligible ages (years): 21.0-55.0, Major Depressive Disorder 55 years of age. MDD currently depressed subjects will meet DSM-V for MDD based on the Mini-International Neuropsychiatric Interview (MINI) (http://www.medicaloutcomes.com/index/mini7fororganizations) with a 17-item Hamilton Depression Rating Scale (HamD17) (Hamilton, 1960) score > 17 Subjects must have failed to enter remission with at least two prior antidepressant medications in the current episode (Vasavada et al., 2016) Must have been free of any medications known to significantly affect brain function for at least ten days prior to enrollment (except fluoxetine, which will require a five-week washout) No unstable medical illness that would prevent completion of participation in the trial (determined as needed from physical examination, ECG, laboratory safety tests, as well as a review of systems) Clinically significant physical abnormalities as indicated by physical examination, hematological laboratory assay, or urinalysis, defined as: hematology and chemistry laboratory tests that are within normal (+/ ) limits with the following exceptions: a) liver function tests (total bilirubin, ALT, AST, and alkaline phosphatase) < 3 x the upper limit of normal, and b) kidney function tests (creatinine and BUN) < 2 x the upper limit of normal A screening ECG that demonstrates anything other than normal sinus rhythm, normal conduction, and no clinically significant arrhythmias History of epilepsy, seizures, or severe head trauma Resting vital signs on any study visit outside of acceptable parameters (i.e., pulse of 60-100 bpm, blood pressures of 90-150 mm Hg systolic, 50 mm Hg diastolic) Any indication of suicidal ideation (e.g. as assessed by the suicidality question on the HamD17or the Columbia Suicide Severity Rating Scale Baseline QT prolongation (QTc> 450 ms): Given that citalopram has been found to be associated with a dose-dependent risk of ECG QT interval prolongation, in order to avoid the potential risk of causing ventricular arrhythmias including Torsades de Pointes, we will participants from the study who exhibit baseline QTc prolongation For women of childbearing age, a positive urine pregnancy test, as well as women who are currently breastfeeding or not using a medically acceptable method of birth control
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Breast Cancer Breast Neoplasms Histologically proven AR and ESFT which includes: Classical, atypical and extraosseous Ewing's sarcoma, primitive peripheral neuroectodermal tumors, peripheral neuroepithelioma, primitive sarcoma of bone, and ectomesenchymoma. Confirmed presence of tumor-specific infusion protein by documented RT-PCR which corresponds to one of the tumor specific peptides available for vaccination. Measurable tumor. No prior or current CNS metastases. PRIOR/CONCURRENT ARM A May be enrolled on the protocol for the first phase in the absence of RT PCR documentation of a tumor-specific fusion protein which corresponds to one of the tumor-specific peptides available for vaccination. However, RT PCR documentation at the time of tumor recurrence must occur prior to administration of immunotherapy. At time of initial tumor diagnosis, prior to any cytoreductive therapy. ARM B Tumor recurrence occurring during or after receiving at least first line cytoreductive therapy for ESFT and AR. No more than two post-recurrence salvage regimens unless peripheral CD4+T cell number is greater than 400 cells per millimeter cubed. At least 6 weeks since any treatments and recovered from all acute toxic effects from time in which immunotherapy will be started for this study. No concurrent estrogen therapy during immunotherapy section of study. Age: 2-25 (at time of initial diagnosis of alveolar rhabdomyosarcoma). Weight: Greater than 15 kg (at time of apheresis). Performance status: ECOG 0-2. Life expectancy: At least 8 weeks. Hematopoietic: ANC greater than 100,000/mm(3). Hemoglobin greater than 9.0 g/dL. Platelet count greater than 50,000/mm(3). Hepatic: Bilirubin less than 2.0 mg/dL (unless related to involvement by tumor). Transaminases less than 3 times normal (unless related to involvement by tumor). Renal: Creatinine less than 1.5 mg/dL or creatinine clearance greater than 60 mL/min. Cardiovascular: No major disorder of cardiovascular system. Cardiac ejection fraction greater than 40%. Pulmonary: No major disorder of pulmonary system. OTHER: Not pregnant or nursing. HIV negative. Hepatitis B or C negative. No patients requiring daily oral corticosteroid therapy. If allergic to eggs, egg products, or thimerosal, or have a history of Guillain-Barre syndrome, ineligible to receive influenza vaccine
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, HIV Infection Children between ages of 4 years and 18 years. Diagnosis of HIV-1 infection as defined by the Centers for Disease Control (CDC) Definition. Availability of a parent or guardian to provide Informed Consent. Child is not critically ill or clinically unstable. No CDC categories N1 and A1 (1994 revised classification for HIV infection in children less than 13 years of age) and the CDC 1993 revised HIV classification and expanded AIDS surveillance definition for adolescents and adults. Non-presence of an active opportunistic infection requiring acute intervention at the time of entry (e.g. CMV, aspergillosis, cryptococcosis, Candida, etc.). Patients receiving treatment for an infection that requires prolonged treatment must have been stable on therapy for at least 30 days prior to study entry. No administration of chemotherapeutic agents, investigational agents or use of immunomodulating agents such as IVIG, corticosteroids, interferons, pentoxifylline, G-CSF/GM-CSF, erythropoeitin, growth hormone and other growth factors within one month of enrollment. None of the following laboratory abnormalities within 2 weeks of study entry: Total WBC count less than 1500/mm(3) or an absolute neutrophil count less than 750/mm(3); Hemoglobin less than 8.0 g/dl; Platelet count less than 75,000/mm(3); Creatinine greater than 2.0 x normal; Creatinine clearance less than or equal to 50 mL/min/m(2); Total bilirubin greater than 2 x normal; SGOT/SGPT greater than 5 x normal; Serum amylase pancreatic isoenzyme greater than 90 U/L (2 x upper limit of normal for adult). Serum amylase pancreatic isoenzyme should be obtained only if total serum amylase is greater than 180 U/L. No history of clinical pancreatitis and/or elevation in serum amylase pancreatic isoenzyme of greater than 180 U/L. No history of peripheral neuropathy of Grade II or greater severity. No previous treatment with ritonavir, indinavir, nelfinavir, nevirapine or stavudine. Patients may have received treatment with ritonavir, indinavir, nelfinavir for less than 4 weeks. Ability to swallow tablets. No child for whom the volume of research blood required for study evaluation exceeds the maximum volume of research blood allowable (3 ml/kg in a single blood withdrawal and 7 ml/kg in a 6-week period). This would be applicable to a child less than 16.5 kg. No patients who refuse or cannot have leukapheresis done. Sexually active post-menarchal females must be willing to use a barrier method of contraception or be willing to remain sexually abstinent
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Cancer Must be 18 years old or greater. ECOG performance status of 0-1. Individuals without a history of cancer are eligible, as are those with a history of cancer. Individuals with a history of cancer (excluding non-melanomatous skin cancer ) will need to submit their pathology slides for review in the Laboratory of Pathology, NCI. Must be able to understand and give informed consent. Life expectancy greater than 6 months. Hgb greater than or equal to 8.0gm/dl, platelets greater than or equal to 100,000/microliters, ANC greater than or equal to 1000/microliters, creatinine less than or equal to 2.0/mg/dl, SGPT and SGOT less than or equal to 147 and 168 U/L, total bilirubin less than or equal to 2 mg/dl (patients with a higher level of bilirubin due to a familial defect in bilirubin metabolism will be considered on an individual basis). No history of breast cancer. No pregnant or breast feeding subjects. Must not be HIV positive. No history of venous thrombosis within the past year. No medical conditions, which, in the opinion of the investigators would jeopardize either the patient or the integrity of the data obtained. No patients who are currently receiving active therapy for neoplastic disorders. However, patients with prostate cancer who are on an LHRH agonist (e.g., Lupron or Zoladex), or who have undergone surgical castration, are eligible for study. No patients who are on estrogen therapy. No patients taking hormonal forms of contraception. No patients with a known soy intolerance
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-18.0, Veno-occlusive Disease Clinical diagnosis of VOD defined by Jaundice (bilirubin ≥ 2 mg/dL) and 2 or more of the following: ascites, weight gain > 5% above baseline weight (see Section 5.2), hepatomegaly, RUQ pain Patients with jaundice and reversal of flow on Doppler examination of the portal vein will be eligible with one of the following: ascites, weight gain > 5% above baseline weight, hepatomegaly, RUQ pain Patients with pre-existing hepatomegaly must have documentation by physical exam or imaging that liver size is increased over baseline at admission Patients who do not meet the in Section 3.1.1 (ie. have two of the major but not three) and have biopsy proven VOD are eligible in the presence of characteristics consistent with severe disease (see below) Patients with concurrent, confounding causes of liver dysfunction clinically evident or evident on ultrasound or other radiographic imaging (such as evidence of biliary ductal dilatation or focal tissue defects) may require biopsy-proven VOD and/or elevated wedged trans-hepatic venous pressure gradient measurement ( ≥10 mm mercury) to be considered eligible. Best medical judgment and further imaging studies can be used to clarify the diagnosis and determine confounding causes of liver dysfunction Severity of VOD defined by For patients addressed by the Bearman model (i.e. within 16 days of SCT and conditioned with either BU/CY, CY/TBI or CBV), there must be a 30% or greater risk of severe VOD (see Appendix B) For all other patients who are not addressed by the Bearman model (ie. beyond 16 days of SCT and/or not conditioned with either BU/CY CY/TBI or CBV), there must be concomitant multi-organ failure defined as presence of one or more of the following Patients receiving concomitant heparin or other anticoagulants unless being used for routine CVL management, fibrinolytic instillation for CVL occlusion, intermittent dialysis or ultrafiltration are excluded. Patients who have received systemic t-PA (concomitant or prior) are excluded Patients with significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells (ie. a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells /24 hours or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss are excluded. These parameters must be reviewed prior to enrollment by the PI or his/her designate Patients with hemodynamic instability as defined below Hemodynamic stability is defined as having no requirement for pressor support OR being able to maintain mean arterial pressure within 1 SD of age-adjusted levels with pressor support (see Appendix D) Patients requiring renal dose dopamine alone (2-5 mcg/kg/min) are eligible without measurement of mean arterial pressure Patients with Grade B-D GVHD, graded according to the IBMTR Severity Index (see Appendix G). Please note patients with Grade B skin only are eligible Patients intubated for documented intrinsic lung disease -- Patients intubated secondary to a mechanical barrier to ventilation, e.g.pulmonary edema or ascites, will be eligible as long as the PaO2/FiO2 ratio is ≥ 300 and/or the oxygen index (OI={MAP x FiO2}÷ PaO2 x 100) is ≤ 25% at the time of enrollment Patients who meet Grade IV common toxicity for neurotoxicity (other than Grade IV confusion and/or delirium), i.e. coma, seizures, toxic psychosis Patients who are currently receiving treatment with another experimental agent
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.082, Cholestasis ENTRY --Disease Characteristics- Severely premature neonates (less than 1000 g at birth and estimated gestational age of no greater than 28 weeks) that require greater than 50% of caloric requirements by parenteral means within 7 days of birth OR Neonates with one or more of the following surgical conditions: Necrotizing enterocolitis Gastroschisis Severe jejunal-ileal atresia within 7 days of diagnosis --Prior/Concurrent Therapy- Biologic therapy: Not specified Chemotherapy: Not specified Endocrine therapy: Not specified Radiotherapy: Not specified Surgery: See Disease Characteristics No other cardiovascular (thoracotomy) or major gastrointestinal surgery (laparotomy) during the newborn period Other: No prior or concurrent ursodeoxycholic acid No concurrent use of extracorporeal life support --Patient Characteristics- Performance status: Not specified Hematopoietic: Not specified Hepatic: Conjugated bilirubin no greater than 1.0 mg/dL No primary or secondary liver disease No hepatic insufficiency as documented by either a biopsy with cirrhosis or elevated prothrombin time without evidence of systemic coagulopathy and no administration of an anticoagulant Renal: No renal failure as indicated by a progressive increase in creatinine levels Other: No hemodynamic instability No documented communicable infection (including infectious hepatitis or HIV) No metabolic pathway defect that is associated with liver dysfunction in the neonatal period including hereditary fructose intolerance, galactosemia due to transferase deficiency, and neonatal tyrosinemia
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Cystic Fibrosis ENTRY --Disease Characteristics-- Cystic fibrosis-associated liver disease, defined by at least one of the following (1) Documented increase in serum concentrations of any of the liver enzymes (at least once in the preceding year) ALT at least twice normal AST at least 1.5 times normal Alkaline phosphatase at least 1.5 times normal GGT at least 1.5 times normal (2) Persistent hepatomegaly of more than 6 months duration defined by percussed liver span greater than 1 SEM for age (3) Splenomegaly, defined as a palpable spleen greater than 2.0 cm below the left costal margin (4) Abnormalities of ultrasound scan (increased size, dishomogeneous echogenicity, nodular liver, irregular margins, splenomegaly) within 6 months prior to study entry Patients enrolled in the first part of the study (objective I) are eligible to participate in the second part (objective II) --Prior/Concurrent Therapy-- At least 3 months since prior ursodiol At least 3 months since treatment with drug with choleretic properties or effects that influence bile acid metabolism --Patient Characteristics-- Hepatic: No decompensated cirrhosis No hepatic neoplasm or cholelithiasis Pulmonary: No significantly impaired pulmonary function with FEV1 less than 50% Other: At least 15 kg body weight No severely compromised clinical or nutritional state
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-6.0, Infant, Newborn Hypoxia-Ischemia, Brain At least 36 weeks gestation Any blood gas (cord, postnatal) done within the first 60 minutes had a pH less than or equal to 7.0 Any blood gas (cord postnatal) done within the first 60 minutes had a base deficit greater than or equal to 16 mEq/L All infants must have seizures or signs of moderate to severe encephalopathy before randomization Inability to randomize by 6 hours of age Presence of known chromosomal anomaly or major congenital anomaly Severe intrauterine growth restriction (weight less than 1800g) All blood gases done within the first 60 minutes had a pH less than 7.15 and a base deficit less than 10 mEq/L Infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist Parents refuse consent Attending neonatologist refuses consent
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.077, Herpes Simplex ENTRY --Disease Characteristics-- Infants diagnosed with herpes simplex virus infection involving the central nervous system with or without evidence of viral dissemination to other organs (i.e., skin, liver, or lungs) HSV-1 or HSV-2 isolated from cutaneous lesions from any site (skin, oropharynx, cerebrospinal fluid (CSF), urine, etc.) OR Must have positive CSF polymerase chain reaction (PCR) if no cutaneous skin lesions are present and viral cultures are negative No infection limited to skin, eyes, or mouth Evidence of CNS involvement includes one or more of the following: Abnormal CSF indices for term infants (WBC greater than 22/mm3 and protein greater than 115 mg/dL) Abnormal CSF indices for preterm infants (WBC greater than 25/mm3 and protein greater than 220 mg/dL) Abnormal neuroimaging study (CT with contrast, MRI with gadolinium, or head ultrasound) Disseminated disease is defined as one or more of the following: SGPT at least 2.5 times upper limit of normal Pneumonia/pneumonitis Necrotizing enterocolitis Disseminated intravascular coagulopathy Birth weight at least 800 grams --Prior/Concurrent Therapy-- No concurrent nursing from a mother who is receiving acyclovir, valacyclovir, or famciclovir for longer than 120 hours or 5 days --Patient Characteristics-- Renal: Creatinine no greater than 1.5 mg/dL Cardiovascular: No prior grade 3 or 4 intraventricular hemorrhage Other: No infants known to be born to HIV positive women
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.077, Herpes Simplex ENTRY --Disease Characteristics-- Infants diagnosed with herpes simplex virus infection limited to skin, eyes, and mouth; HSV-1 or HSV-2 isolated from cutaneous lesions, conjunctivae, or oropharynx (presence of skin lesions not required); normal CSF indices: WBC less than 22/mm3 and protein less than 115 mg/dL for term infants OR WBC less than 25/mm3 and protein less than 220 mg/dL for preterm infants; no evidence of CNS involvement by CT with contrast, MRI with gadolinium, or head ultrasound; no visceral dissemination (normal liver function tests, normal chest x-ray, etc.); negative CSF PCR result Birth weight at least 800 grams --Prior/Concurrent Therapy-- No concurrent nursing from a mother who is receiving acyclovir, valacyclovir, or famciclovir for longer than 120 hours or 5 days; no prior prophylactic acyclovir for risk of herpes simplex virus infection --Patient Characteristics-- Renal: Creatinine no greater than 1.5 mg/dL Cardiovascular: No prior grade 3 or 4 intraventricular hemorrhage Other: No infants known to be born to HIV-positive women
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Adenocarcinoma of the Pancreas Stage II Pancreatic Cancer Stage III Pancreatic Cancer Histologically confirmed unresectable, locally advanced adenocarcinoma of the pancreas Residual disease after resection (R1 or R2, microscopic or macroscopic) allowed No metastases in major viscera No peritoneal seeding or ascites Biliary or gastroduodenal obstruction must have drainage before starting study therapy Radiographically assessable disease encompassable within a single irradiation field (15 by 15 cm maximum) Performance status Zubrod 0-1 Granulocyte count at least 1,800/mm^3 Platelet count at least 100,000/mm^3
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.019, Neonatal Jaundice Hyperbilirubinemia Term or near term neonate Elevated serum bilirubin Failing phototherapy Requires exchange transfusion Family refuses exchange transfusion on religious grounds
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 18.0-70.0, DS Stage I Plasma Cell Myeloma DS Stage II Plasma Cell Myeloma DS Stage III Plasma Cell Myeloma Refractory Plasma Cell Myeloma Smoldering Plasma Cell Myeloma Patients must have active multiple myeloma requiring treatment (Durie-Salmon stage >= 1) and have stable disease or be responsive to at least 2 months of any induction therapy; patients with smoldering myeloma are not eligible unless the disease has progressed to >= stage 1 No more than 12 months of any prior therapy, including CC-5013 and thalidomide Within 12 months of initiation of induction therapy No prior progression after initial therapy; in addition, no more than two regimens will be allowed excluding dexamethasone alone No prior peripheral blood, bone marrow, or solid organ transplant Patients must have peripheral blood stem cell collection of >= 2 x 10^6 cluster of differentiation (CD)34+ cells/kg (patient body weight) and preferably 5 x 10^6 cells/kg (patient body weight); stem cells may be collected at any time prior to transplant; peripheral blood stem cell collection may occur before or after registration Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 Patients must have diffusing capacity of the lung for carbon monoxide (DLCO) > 50% predicted with no symptomatic pulmonary disease Patients must have left ventricular ejection fraction (LVEF) >= 40% by multi gated acquisition scan (MUGA) or echocardiogram Patients must not have uncontrolled diabetes mellitus
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-36.0, Hyperbilirubinemia, Neonatal Jaundice, Neonatal Infant, Newborn Infant, Low Birth Weight Infant, Small for Gestational Age Infant, Premature 1000 grams birth weight 36 hours postnatal age Terminal condition (pH <6.8 for >2 hours OR persistent bradycardia, heart rate <100 bpm, associated with hypoxia for >2 hours] Prior use of phototherapy Major congenital anomaly Hydrops fetalis or severe hemolytic disease diagnosed in-utero Overt congenital nonbacterial infection Parental refusal or inability to provide consent Attending physician refusal Parents who are considered unlikely to return for follow-up evaluation
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1
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.008, Preterm Birth Preterm newborns with birth weight equal to or less than 1500 grams and gestational age equal to or less than 32 weeks Mother willing to participate in study Major congenital malformations Congenital infectious diseases Mother not willing to participate in study
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1
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 15.0-38.0, Malaria A pregnant woman who has either a positive blood smear for P.falciparum with at least 1000 asexual parasites/uL in an asymptomatic woman or any of the following symptoms within 2 days prior to consultation history of fever headache vomiting chills/rigors and/or any of the following signs: temperature >37.50C and <39.50C, Hb>5 and <9 g/dl together with P.falciparum parasitaemia at any density and (in both cases) the following: 1. Has no criterion (see below); 2. Is 14-34 weeks pregnant on the day of attending the ANC clinic or OPD; 3. Has a viable foetus, defined by presence of foetal heartbeat by sonicaid or pinnard (foetal heartbeat is not heard until 14 weeks); 4. Is able to take study drugs by the oral route; 5. Is able to attend stipulated days for follow up clinic and provide specimens; 6. Gives informed written or witnessed verbal consent to participate by herself, and also through her parent/guardian if aged <15 years (in conformity to Tanzania Law) Severe and complicated forms of malaria (as defined by WHO, 1996); 2. Pregnancy in the first trimester; 3. A mixed plasmodial infection; 4. Complicated pregnancy, e.g. signs/symptoms of toxaemia of pregnancy; 5. 23 or more abortions or stillbirths; 6. Presence of concomitant disease masking assessment of the response to treatment ; 7. An intake of drugs contraindicated in pregnancy, e.g. tetracycline, cotrimoxazole or a macrolide antibiotic; 8. An intake of drugs with effective antimalarial activity within the last 2 weeks. 9. Significantly abnormal baseline haematology (except anaemia) or clinical chemistry parameters, e.g. laboratory evidence of renal impairment (serum creatinine >2 mg/dl) or of hepatitis (alanine aminotransferase [ALT] >5 times upper limit of normal); 10. Previous participation in the study: Women having a second episode of malaria after completing the 28-day follow up will have details recorded and offered quinine but not be re-enrolled. 11. Multiple gestation pregnancies, eg twins 12. Mother aged 38 years or above
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Pre-eclampsia A subject with a diagnosis of severe preeclampsia will be eligible for if she meets the following 1. In the opinion of the investigator delivery is considered to be probably required within a 72 hour time period and, therefore, corticosteroid administration is needed. 2. Meets both American College of Obstetricians (ACOG) for preeclampsia (modified to limit selection to patients with the required severity) A systolic blood pressure of 140 mm Hg or higher or a diastolic blood pressure of 90 mm Hg or higher occurring after 20 weeks of gestation in a woman whose blood pressure has previously been normal Proteinuria, with excretion of 0.3 g or more of protein in a 24-hour urine specimen or a urine dipstick reading of 1+ or more. 3. Meets at least one of the following ACOG for severe preeclampsia (modified to limit selection to patients with the required severity) . Proteinuria of 5 grams or higher in a 24-hour specimen or 3+ or greater on 2 random urine samples collected at least 4 hours apart A systolic blood pressure of 160 mm Hg or higher or a diastolic blood pressure of 110 mm Hg or higher on two occasions six or more hours apart in a pregnant woman who is on bed rest Oliguria, with excretion of less than 500 ml of urine in 24 hours or average of ≤ 25 ml/hour over a 3 hour period Pulmonary edema Impairment of liver function [AST(SGOT) > 72 U/L or ALT(SGPT) > 72 U/L or LDH > 600 U/L or Total Bilirubin >1.2 mg/DL)] Visual or cerebral disturbances Decreased platelet count (≥50,000/mm3 and ≤ 100,000/mm3). 4. Has a fetal gestational age of 23 5/7 to 34 weeks Is in need of immediate delivery as soon as clinically appropriate 2. Eclampsia 3. Significant antecedent obstetrical problems which may interfere with study assessments or safe participation in the study 4. Evidence of non-reassuring fetal well being 5. Evidence of lethal fetal anomaly 6. Antecedent hypertension (hypertension secondary to preeclampsia, treated or untreated is allowed) 7. Antecedent renal, hepatic, or autoimmune disease 8. Medical or psychiatric disorder which is unstable or which might interfere with study assessments or safe participation in the study 9. Evidence on medical history/evaluation of use of or need for digitalis-like products currently or in the future 10. History of a severe allergic reaction to previous medication, severe asthma, or atopy. (Patients with a history of allergic reactions to antibiotics, papain, chymopapain, or other papaya extracts may be more susceptible to allergic reactions to Digibind®) 11. Prior use of antibodies/FAB fragments from sheep (e.g. Digibind®, DigiFab, CroFab) 12. Serum creatinine ≥ 1.5 mg/dl 13. Platelet count <50,000/mm3 14. Patient intends to breast feed and does not agree to wait for a minimum of seven days after the last Digibind® dose (a breast pump would be used for this seven day period) 15. Inability to understand and provide informed consent
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Prenatal Exposure Delayed Effects Multiple Sclerosis Pregnancy Key Have been exposed to within approximately 1 week of conception or during the first trimester of pregnancy Provide sufficient information to determine that the pregnancy is prospectively registered (i.e., the outcome of pregnancy must be unknown prospectively) Provide verbal consent to participate in the Registry Verbally provide contact information for herself, her HCP, and the infant's HCP (if applicable). NOTE: Other protocol defined inclusion/
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-18.0, Liver Transplantation Subjects who meet all of the following are eligible for this study: 1. Male or female patients, not older than 18 years old. 2. Primary liver transplantation 3. Patient is capable of understanding the purpose and risks of the study and has been informed both orally and in writing and has given informed consent Subjects who meet one or more of the following are not eligible for this study: 1. Female patients who are pregnant or are breast feeding 2. Patients > 18 years old 3. Combined liver-kidney transplantation 4. Recipient of second liver graft 5. Patients are allergic, hyper-sensitive or intolerant to HCO-60 or structurally related compounds, macrolide antibiotics or tacrolimus. 6. Patients with known HIV-anamnesis 7. Patient requires ongoing dosing with a systemic immunosuppressive drug at study entry for another indication than the prophylaxis of liver graft rejection 8. Patient has significant, uncontrolled concomitant infections and/or severe diarrhoea, vomiting, or active peptic ulcer. 9. Patient is participating or has participated in another clinical study and/or is taking or has been taking an investigational drug in the past 28 days. 10. Other reasons which depend on the assessment of the physician (no MMF will be given to patients with severe persistent hypersplenism (WBC < 3.500/ml, platelets < 50.000/ml)
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-0.25, Stress Sleep All infants born at 28 weeks gestation or who are at least 28 weeks corrected age are eligible to participate. Only those in isolettes (incubators) will be included. Once a baby has been transitioned to a bassinet or crib, the baby will be pulled from the study. In order to be included, these infants must be in stable condition (without serious active medical issues) as decided by their doctor and must be breathing without the use of a ventilator will a history of intraventricular hemorrhage involving the white matter (as diagnosed by head ultrasound), as well as those infants born with severe congenital deformities
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1
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-999.0, Carcinoma, Non-Small-Cell Lung Patients must have histologic evidence of NSCLC. Patients with bronchoalveolar type of are not eligible. 2. Should a second malignancy be present or discovered, subjects will only be eligible if the is determined by the PI to be the more life-threatening disease compared to the other malignancy in regards of life-expectancy. 3. All patients must have locally advanced disease (stage III A or III B) or metastatic (stage 4). Other stages are not eligible. 4. Performance status of 0 -1 (ECOG Criteria). 5. Patients should have an absolute granulocyte count > 1000/mm3 and a platelet count > 80,000/mm3. 6. Patients should have adequate hepatic function as indicated by a serum bilirubin < upper limit of normal (ULN); ALT and AST <2.5 ULN if alkaline phosphatase is < ULN. Alkaline phosphatase may be up to 4 x ULN if transaminases are < ULN. However, patients who have both transaminase elevation > 2 x ULN and alkaline phosphatase > 2.5 x ULN are not eligible for this study. 7. Subjects with with severe renal impairment (creatinine clearance below 30 mL/min [Cockroft and Gault]) are ineligible. 8. Patients should have at least a predicted FEV1 of 30%. 9. Patients with active ischemic heart disease (NYHA Class III or IV), congestive heart failure, symptomatic arrythmias, or a recent history of a myocardial infarction are excluded. 10. Patients with medically uncontrollable hypercoagalbility syndromes are not eligible. Patients who are on therapeutic anticoagulation are not excluded. 11. Patients with pre-existing serious adverse effects to 5-FU are not eligible for this study. Specifically, subjects with prior unanticipated severe reaction to fluoropyrimidine therapy or known hypersensitivity to 5-fluorouracil. 12. Patients with medical contraindications to Taxanes are not eligible. 13. No other serious concurrent medical illness or active infection which would jeopardize the ability of the patient to receive with reasonable safety the chemotherapy and surgery program outlined in this protocol is allowed. 14. Subjects where studies or clinical examination demonstrates lack of physical integrity of the upper gastrointestinal tract, inability to swallow tablets or those who have malabsorption syndrome are not eligible. 15. Subjects who have had an organ allograft are not eligible. 16. Signed informed consent: each patient must be aware of the neoplastic nature of his/her disease and willingly consent after being informed of the procedure to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts. 17. Pregnant women and nursing mothers are ineligible. Eligible patients of reproductive age should use contraception. Woman of childbearing potential with either a positive or no pregnancy test at baseline. Woman of childbearing potential not using a reliable and appropriate contraceptive method. (Postmenopausal women must have been amenorrheic for at least 12 months to be considered of non-childbearing potential). 18. Sexually active males unwilling to practice contraception during the study will not be eligible. 19. Patients must be at least 18 years old. 20. Known HIV positive patients will not be eligible. 21. Patients with prior treatment of Taxotere, Xeloda or 5FU will not be eligible
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0
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The patient is a 3-day-old female infant with jaundice that started one day ago. She was born at 34w of gestation and kept in an incubator due to her gestational age. Vital signs were reported as: axillary temperature: 36.3°C, heart rate: 154 beats/min, respiratory rate: 37 breaths/min, and blood pressure: 65/33 mm Hg. Her weight is 2.1 kg, length is 45 cm, and head circumference 32 cm. She presents with yellow sclera and icteric body. Her liver and spleen are normal to palpation. Laboratory results are as follows: Serum total bilirubin: 21.02 mg/dL Direct bilirubin of 2.04 mg/dL AST: 37 U/L ALT: 20 U/L GGT: 745 U/L Alkaline phosphatase: 531 U/L Creatinine: 0.3 mg/dL Urea: 29 mg/dL Na: 147 mEq/L K: 4.5 mEq/L CRP: 3 mg/L Complete blood cell count within the normal range. She is diagnosed with neonatal jaundice that may require phototherapy.
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eligible ages (years): 0.0-6.0, Hyperbilirubinemia Erythroblastosis, Fetal All newborns with a gestational age equal or higher than 32 weeks, with a Rh (D) positive blood type, children of sensitized Rh (D) negative mothers, regardless if they were submitted or not to an intra-uterus transfusion Newborns in serious condition, hydropic, hemodynamically instable or with indication for exchange transfusion at birth. The indications for exchange transfusion at birth are: presence of bilirubin in the umbilical cord higher or equal to 4mg%; hydrops, cardiac insufficiency secondary to severe anemia
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0
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