Clinical Trial Search

Primary: Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score — -3.8; -1.8; 0.0 score on a scale

Primary: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) — -1.65; -3.40 score on a scale — p=0.0514

Primary: Safety (Adverse Events) — 8; 6; 0; 0 Participants

Primary: The Number of Study Participants Who Safely Tolerated Somatokine — 6; 9 participants

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…

Primary: Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance — 17.44; 7.25 Meters

Primary: Oxygen Consumption — 72; 102 mL oxygen/min/m^2

Primary: Adult Height Relative to General Population — -0.34; -0.6 Standard Deviation Score (SDS) units

Primary: Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700 — 0 Participants

Primary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters

Primary: Number of Participants With Adverse Events — 2; 1; 5; 4 Participants

Primary: 12-month Change in Myocardial Strain — 2.2; 1.0 percent change in heart dimension

Primary: Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month) — 4.8 units on a scale

Primary: Fasting Glucose — 93; 102 mg/dL

Primary: Forced Vital Capacity — 1.4; 1.4; 1.5 liters — p=0.3904