Clinical Trial Search

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 Treatment-Emergent Adverse Events — 14; 9; 10 Participants

Primary: Muscle, Nerve, or Vascular Damage — 16 participants

Primary: Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil — 1.05; 4.75; 2.15; 3.27 ng/mL

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

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

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

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

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

Primary: Time to Sedation Score of 3-4 — 3.7; 2.8 minutes

Primary: Fasting Glucose — 93; 102 mg/dL

Primary: Number of Participants With Adverse Events Related to AVI-4568 — 0; 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: 12-month Change in Myocardial Strain — 2.2; 1.0 percent change in heart dimension

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

Primary: Number of Unanticipated Grade III or Higher Treatment-Related Toxicities — 0; 0 events

Primary: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks — 0.55; 0.86 AU — p=0.0434

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 Dose Limiting Toxicity (DLT) Adverse Events as Assessed by 21 CFR 312.32. — 0 Number of Events

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

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

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

Primary: Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis — -0.13; -1.75; -0.75; -1.14 Scores on a scale…

Primary: Mean Change From Baseline in Muscle Function Using the 6 Minute Walking Distance — -10.98; -19.93; 16.12 Meters — p=0.554

Primary: Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48 — -52.65; -42.32 Meters — p=0.415

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

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

Primary: Percent Change in Range of Motion From Baseline to 180 Days — 67; 33 percentage of change

Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 — 2.57; 1.92 scores on a scale — p=0.2441

Primary: Plasma Follistatin