65,437 trials
Myotonic Dystrophy, Type 1 (DM1) · Myotonic Dystrophy
Primary: Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo — 0; 0; 1; 1…
Colorectal Cancer
Primary: Phase 1b: Number of Participants With Incidence of Dose Limiting Toxicities (DLTs): Cycle 1 — 0; 1; 1; 1 Participants
Postmenopausal Women
Primary: Resting Aortic Blood Pressures — 113.3; 116.9 mmHg
Non-small Cell Lung Cancer
Primary: Overall Survival — 11.79; 8.25 Months
Q Fever · Fatigue Syndrome, Chronic · Coxiella Infection
Primary: Checklist Individual Strength (CIS) — 31.6; 40.8; 37.8 score on a scale
Respiratory Tract Infections
Primary: Percentage of Subjects With Clinically Symptomatic Respiratory Illness — 125; 134 Participants
Rosacea
Primary: Percentage of Subjects With Treatment Success at All Time Points 3, 6, 9 and 12 Hours Post-application on Day 29. Treatment Success Was Defined as Having a Clinician…
Colon Polyps
Primary: Number of Participants With Delayed Bleeding Complications — 16; 33 Participants
Therapeutic Equivalency
Primary: Maximum Observed Plasma Concentration (Cmax) — 15.49; 17.35 ng/mL — p=0.3008
Advanced Cancer
Primary: Disease-free Survival (DFS) — 22.41; 11.04 Months — p=0.0003
Alagille Syndrome
Primary: Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA) — 223.62; 157.35 μmol/L — p=0.1157
Health Behavior · Parenting · Social Acceptance
Primary: Enrollment in Evidence-Based Parenting Program — 27; 12 Participants — p=0.006
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
Sleep Disordered Breathing · Sleep Apnea · Paroxysmal Atrial Fibrillation
Primary: Change in AF Burden — 5.6; 5.0; 4.1; 4.3 percentage of time in AF — p=0.520
Gastrointestinal Stromal Tumors (GIST) · Other Relapsed or Refractory Solid Tumors
Primary: Part 1: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Avapritinib — 0; 0; 0; 0 patients with event(s) of dose-limiting