Clinical Trial Search

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

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

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

Primary: Patient-reported Stiffness on the IVR — 2.53; 4.21; 1.60; 5.27 units on a scale — p=<0.001

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

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 From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756

Primary: Incidence of Treatment-Emergent Adverse Events — 14; 9; 10 Participants

Primary: Number of Participants With Adverse Events Related to AVI-4568 — 0; 0 Participants

Primary: Number of Participants With a Decrease in Cramp Duration — 2; 0 participants

Primary: Change From Baseline in 6MWD at Week 48 — -60.67; -47.69 meters — p=0.213

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants

Primary: Number of Subjects With an Adverse Event. — 1; 0; 0; 0 participants

Primary: Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts — 1; 1…

Primary: Percent Change From Baseline in Dystrophin Level at Week 40, as Measured by ECL — 6.559 percent change

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

Primary: Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline — 3.4; -5.1 meters — p=0.53

Primary: Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) — 0; 0; 0; 0 Participants

Primary: Age of Participants When They Failed to Walk — NA; 14.4; 14.4; 11.1 Years

Primary: Bone Mineral Density in FSHD — -0.4 z-score

Primary: Count of Adverse Events Reported — 109; 52 adverse events reported

Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88

Primary: Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention — 5.45; 6.61; 6.64; 6.57 score on a scale — p=.211

Primary: Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy — 3; 5 Participants

Primary: Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical…

Primary: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population — -81.83; -90.09 meters — p== 0.3626

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 4; 5; 3; 3 Participants

Primary: Note Errors (Related to Errors in Duration) — 8.20; 5.79 msec — p==0.06