Clinical Trial

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

Study acronym: ACHIEVE
Recruiting Phase 1/2
View on ClinicalTrials.gov →
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.
Protocol Amendment History 1 change
notable Trial sites expanded: 17 -> 20 locations 2026-05-13
Trial Details
NCT Number NCT05481879
Lead Sponsor Dyne Therapeutics
Conditions Myotonic Dystrophy Type 1 (DM1)
Enrollment 116 participants
Start Date 2022-09-05
Primary Completion 2029-07 (estimated)
Study Completion 2029-07 (estimated)
Updated on ClinicalTrials.gov 2026-05-12