HomeTrialNCT05481879
🔬NCT05481879Phase 1Phase 2RECRUITING

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

Study Summary

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment116 participants
Primary Completion
Lead SponsorDyne Therapeutics
Data Retrieved2026-04-11 04:12:12.434007+00:00
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