clinical trial · NCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1)DM1Myotonic DystrophySteinert DiseaseSteinertzeleciment basivarsen (DYNE-101)Placebo
brief summary
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).
started
Apr 1, 2026
primary completion
Jul 1, 2028
completion
Jan 1, 2029
last updated
Apr 21, 2026
detailed description
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks).
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
official title
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol