Phase 2 N=10 Mepolizumab As a Steroid-sparing Treatment Option in the Churg Strauss Syndrome
Churg Strauss Syndrome
Primary: Number of Participants With Indicated Side Effects — 3; 1; 0; 1 Participants
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Phase 3 N=100 Long-term Access Program (LAP) of Mepolizumab for Subjects Who Participated in Study MEA115921
Churg-Strauss Syndrome · Eosinophilic Granulomatosis With Polyangiitis
Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 98; 38 Participants
N/A N=118 Mepolizumab Long-term Study to Assess Real World Safety and Effectiveness of Eosinophilic Granulomatosis With Polyangiitis (EGPA) in Japan
Churg-Strauss Syndrome · Eosinophilic Granulomatosis With Polyangiitis
Primary: Number of Participants With Any Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESI) — 69; 26; 42 Participants
Phase 3 N=20 Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).
Photosensitive Epilepsy
Primary: % Change in Frequency of Handwaving Episodes — -66.2 % change in hand waving episodes — p=<0.001
Phase 3 N=31 Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
Nephrotic Syndrome
Primary: Number of Participants Experienced a Relapse of Nephrotic Syndrome — 14; 15 Participants
N/A N=1,504 Non-interventional Observational Study on the Influence of Pramipexole on Sensory Symptoms of Restless Legs Syndrome (RLS)
Restless Legs Syndrome
Primary: Summary of Change From Baseline in Total Sum of McGill Pain Questionnaire to Visit 3 — -14.48 Scores on scale
Phase 4 N=36 Oral Contraceptives in the Metabolic Syndrome
Metabolic Syndrome X · Insulin Resistance · Obesity
Primary: Changes in Insulin Sensitivity Associated With Oral Contraceptive (OC) Use Compared Among (1) Obese Women and (2) Lean Women — 4.36; 6.62; 3.82; 8.23 mIU/L
Phase 1 N=12 Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Rett Syndrome
Primary: Adverse Events — 4; 4; 2; 17 Adverse events
N/A N=473 Improving Medication Adherence in the Alabama Black Belt
Diabetes Mellitus · Medication Adherence
Primary: Change in Self Reported Medication Adherence — 0.78; 0.74; 0.53; 0.62 Scores on a scale — p=0.22
N/A N=10 Transcranial Magnetic Stimulation for Children With Tourette's Syndrome
Tourette Syndrome
Primary: Yale Global Tic Severity Scale — 64.40; 26.40 units on a scale
Phase 4 N=39 Efficacy and Safety of Memantine Hydrochloride in Enhancing the Cognitive Abilities of Young Adults With Down Syndrome
Down Syndrome
Primary: Changes in Neuropsychological Measures From Baseline to End of Study — -0.22; -0.84; 0.56; 0 units on a scale — p=0.403
Phase 3 N=40 Effects of Losartan Versus Atenolol on Aortic and Cardiac Muscle Stiffness in Adults With Marfan Syndrome
Marfan Syndrome
Primary: Aortic Biophysical Properties - Pulse Wave Velocity — -1.15; -0.22 change in meters/second
N/A N=51 Impact of Consumption of Beta-glucans on the Intestinal Microbiota and Glucose and Lipid Metabolism
Metabolic Syndrome · Dyslipidemia · Obesity, Abdominal
Primary: Total Cholesterol Levels — 5.8; 5.9 mmol/L
Phase 2 N=19 Mifepristone Effects on Glucose Intolerance in Obese/Overweight Adults
Endocrine Disease · Diabetes
Primary: Change in Insulin Sensitivity Index — 1.49; 1.41 min-1·μU·ml-1 — p=0.60
Phase 4 N=15 Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
Treatment Resistant Nephrotic Syndrome
Primary: Significant Reduction in Proteinuria to Remission Levels During the Treatment Period (Includes Complete and Partial Remission, as Defined in the Outcome Measure…