17,261 trials
Menkes Disease · Occipital Horn Syndrome
Primary: Treatment Related Adverse Events as Assessed by CTCAE v4.0 — 10; 6 adverse events
Growth Hormone Deficiency
Primary: Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer — 0.098 cm/year
Alport Syndrome
Primary: Percentage of Patients With Serious Adverse Events (SAEs) — 1; 0 Participants
Acid Sphingomyelinase Deficiency
Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 3; 0 Participants
Lung Transplant · CMV
Primary: Occurrence of CMV Infection or Disease During Prophylaxis — 0 Participants
Diabetes Mellitus, Type 2
Primary: Change From Baseline in Glycosylated Haemoglobin (HbA1c) — -2.3; -2.2; -2.2; -2.3 Percentage point of HbA1c — p=<0.0001
Severe Malaria
Primary: Percentage of Participants Achieving at Least 90% Reduction in Plasmodium Falciparum (P. Falciparum) at 12 Hours — 80.0; 93.0; 39.3 percentage of participants
Rheumatoid Arthritis
Primary: Safety/Tolerability (Adverse Events) — 9; 9 Participants
Lung Cancer Non Small Cell · Head and Neck Squamous Cell Carcinoma · Urothelial Carcinoma Bladder
Primary: ORR — 15; 8; 2 Participants
Metastatic BRCA Hereditary Breast Carcinoma · Metastatic Breast Carcinoma · Metastatic Malignant Neoplasm in the Brain
Primary: Progression-free Survival (PFS) — 6.4; 6.2; 4.2; 5.9 months
Postural Orthostatic Tachycardia Syndrome · Chronic Orthostatic Intolerance · Syncope
Primary: Blood Pressure — 101; 107; 64; 68 mmHg
Metastatic Pancreatic Adenocarcinoma
Primary: Phase I: Determine the Recommended Phase II Dose (RP2D) — 300 mg
Urticaria
Primary: Effect of ARS-1 Versus Placebo Based on a Patient Reported Pruritus/Hive Score — 1.1; 0.8; 1.8; 0.4 UAS score 0 to 3
Acute Myeloid Leukemia
Primary: Duration of Remission Response to Azacitidine and Venetoclax Treatment + Maintenance Therapy — 12.9 months
Advanced Cancer
Primary: Number of Participants With Grade 3/Grade 4 Laboratory Test Results — 0; 0; 0; 0 Participants