A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 in Participants With Myotonic Dystrophy Type 1

What this Trial Is About

This research aims to evaluate the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) in people with myotonic dystrophy type 1 (DM1). The study is a phase 3 clinical trial designed to better understand how this treatment works for DM1, a genetic condition confirmed by molecular testing with a specific genetic marker. It is sponsored by Dyne Therapeutics and includes a detailed monitoring process to ensure participant safety throughout the trial. The study includes three main periods: a screening period lasting up to 8 weeks, a 48-week placebo-controlled treatment period, and a 24-week long-term extension period. Participants will be randomly assigned to receive either DYNE-101 or a matching placebo through intravenous infusion once every 8 weeks during the placebo-controlled phase. After this phase, all participants will receive DYNE-101 every 8 weeks for the long-term extension. Participants will be involved in regular assessments including physical tests like the 5 Times Sit-To-Stand and 10-Meter Walk/Run tests, muscle strength evaluations, and patient and clinician global impressions of change and severity. Blood samples will be collected to study the treatment's behavior in the body and check for immune responses. Safety and tolerability will be closely monitored by an independent committee. The full participation period can last up to about 80 weeks including all study phases.

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