Phase 3 Evaluation of DYNE-101 Infusion for Treating Myotonic Dystrophy Type 1 with Placebo Comparison and Extension Period

What this Trial Is About

This research aims to evaluate the effectiveness, safety, and tolerability of zeleciment basivarsen DYNE-101 in treating participants with myotonic dystrophy type 1 DM1. The trial is a Phase 3 interventional study sponsored by Dyne Therapeutics and includes a comparison with a placebo. The study includes an independent committee that regularly reviews safety data to ensure participant well-being. Participants will be randomly assigned to receive either DYNE-101 or a matching placebo through intravenous infusion every 8 weeks during a 48-week placebo-controlled period. Following this, all participants will enter a 24-week long-term extension period where those initially on placebo will begin receiving DYNE-101, while those already on DYNE-101 will continue treatment on the same schedule. During the study, participants will undergo various assessments including physical performance tests like the 5 Times Sit-To-Stand and 10-Meter WalkRun Test, muscle strength measurements, clinician and patient global impressions, and health indexes specific to DM1. Blood samples will be collected to analyze drug concentration and antibody levels. The total participation spans up to approximately 80 weeks, including screening, treatment, and follow-up, with ongoing safety monitoring throughout.

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1