Status: Recruiting
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
Participants needed: 150
Trial details
Phase: Phase 3Age: 16+Biological sex: AllType: InterventionalSponsor: Dyne TherapeuticsUpdated: Jun 30, 2026Locations: 14
Eligibility criteria
Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size... [+2]
A known diagnosis of congenital DM1. [+2]