Study of DYNE-101 Given by IV to Adults With Myotonic Dystrophy Type 1 to Evaluate Safety, Tolerability, and Effects

What this Trial Is About

Researchers are evaluating the safety and tolerability of multiple intravenous doses of DYNE-101 in adults with Myotonic Dystrophy Type 1 DM1, a condition characterized by muscle symptoms and myotonia. This randomized, placebo-controlled study involves both multiple-ascending dose and dose expansion groups. The study is designed to better understand how DYNE-101 behaves in the body and its effects on muscle function and myotonia severity. The study includes four periods a Screening Period lasting up to 8 weeks, a Placebo-Controlled Period of 24 weeks where participants receive either DYNE-101 or placebo by IV infusion every 4 or 8 weeks, a Treatment Period of 24 weeks where all participants receive DYNE-101, and a Long-Term Extension Period of up to 168 weeks where participants continue receiving DYNE-101. Dosing schedules vary between every 4 weeks Q4W and every 8 weeks Q8W. Participants will undergo various assessments throughout the study, including tests of muscle function such as hand grip relaxation time, walkrun tests, and stair climbing, as well as muscle tissue analysis for myotonia and other biomarkers. Researchers will monitor safety by tracking adverse events and drug levels in the blood. The study lasts up to about 4 years, including long-term follow-up to evaluate the effects and safety of DYNE-101 over time.

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1