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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
2026-05-14
Completion Date
2029-01
Last Updated
2026-07-14
Healthy Volunteers
No
Interventions
zeleciment basivarsen (DYNE-101)
Administered by IV infusion
Placebo
Administered by IV infusion
Locations (18)
UCSD - Altman Clinical and Translational Research Institute
La Jolla, California, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
IU Health Neuroscience Center
Indianapolis, Indiana, United States
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, 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
UZ Leuven
Leuven, Belgium
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
Copenhagen, Denmark
CHU de Nantes - Hôtel-Dieu
Nantes, France
Pitié-Salpêtrière Hospital
Paris, France
CHU de Toulouse - Hôpital Pierre-Paul Riquet
Toulouse, France
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
Munich, Bavaria, Germany
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
Berlin, State of Berlin, Germany
Centro Clinico Nemo Milano, Fondazione Serena ETS
Milan, Milano, Italy
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, Japan
Yamaguchi University Hospital
Ube-Shi, Yamaguchi, Japan
Hospital Universitario Infanta Sofía
San Sebastián de los Reyes, Madrid, Spain
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London, England, United Kingdom