12,372 trials
Fabry Disease
Primary: Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) — 5 Participants
Cystic Fibrosis
Primary: Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 129; 31…
Asthma
Primary: Annual Asthma Exacerbation Rate in Adult and Adolescent Patients With Uncontrolled Asthma — 0.93; 2.10 events per year — p=<0.001
Ulcerative Colitis (UC)
Primary: Percentage of Participants Who Achieved Clinical Remission Per Adapted Mayo Score at Week 8 — 33.5; 4.1 percentage of participants — p=<0.001
Congenital Bleeding Disorder · Haemophilia A
Primary: Number of Adverse Events Reported — 58; 444; 8 Events
Hepatitis C
Primary: Number of Participants With Undetectable HCV RNA at 12 Weeks Post End of Treatment (SVR12) Following SOF/VEL for 6 Weeks as Compared With 12 Weeks in People With Recent…
Idiopathic Hypersomnia
Primary: Change in Epworth Sleepiness Scale (ESS) Score — 0.7; 7.4 score on a scale
Chemotherapy-induced Thrombocytopenia
Primary: Percentage of Subjects Who do Not Require Platelet Transfusion, Dose Reduction in Chemotherapy by 15%, or Chemotherapy Delay by >=4 Days — 57; 29 Participants — p=0.7186
Diabetic Retinopathy · Diabetic Macular Edema
Primary: Development of PDR and/or DME (Whichever Came First) — 27; 75 Eyes
Coronary Artery Disease (CAD)
Primary: Absolute Change From Baseline in Minimum FCT — 21.5; 42.7 µm — p=0.015
Fanconi Anemia
Primary: Number of Participant With Neutrophil Recovery — 78 Participants
Caries
Primary: Caries Arrest of Teeth in the SDF Group Measured by Change in Size — 10 Participants
Malaria
Primary: Percentage of Participants With PCR-Corrected ACPR on Day 28 — 97.6; 98.8 percentage of cured subjects — p=<0.0001
Sars-CoV2 · COVID
Primary: Composite Outcome of Arterial Thromboembolic Events, Venous Thromboembolic Events and All-cause Mortality at Day 30 ± 2 Days. — 25; 31 Participants
Acquired Thrombotic Thrombocytopenic Purpura
Primary: Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events — 37.9; 8.2 percentage of participants