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Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Sponsor: Dyne Therapeutics
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).
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
Key Details
Gender
All
Age Range
16 Years - Any
Study Type
INTERVENTIONAL
Enrollment
150
Start Date
2026-04
Completion Date
2029-01
Last Updated
2026-05-06
Healthy Volunteers
No
Interventions
zeleciment basivarsen (DYNE-101)
Administered by IV infusion
Placebo
Administered by IV infusion
Locations (5)
Rare Disease Research, LLC
Atlanta, Georgia, United States
Rare Disease Research, LLC
Hillsborough, North Carolina, United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, Japan
Yamaguchi University Hospital
Ube-Shi, Yamaguchi, Japan