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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
2026-08-18
Healthy Volunteers
No
Interventions
zeleciment basivarsen (DYNE-101)
Administered by IV infusion
Placebo
Administered by IV infusion
Locations (34)
UCSD - Altman Clinical and Translational Research Institute
La Jolla, California, United States
UCLA Medical Center (Department of Neurology)
Los Angeles, 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
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States
Virginia Commonwealth University - Biotech One
Richmond, Virginia, United States
UZA
Edegem, Belgium
UZ Leuven
Leuven, Belgium
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
Copenhagen, Denmark
AP-HP, Hôpital Raymond-Poincaré
Garches, France
CHU Marseille - Hôpital de la Timone (APHM)
Marseille, France
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
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen
Bonn, North Rhine-Westphalia, Germany
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
Berlin, State of Berlin, Germany
Centro Clinico Nemo Brescia, Fondazione Serena ETS
Gussago, Brescia, Italy
Centro Clinico Nemo Milano, Fondazione Serena ETS
Milan, Milano, Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile
Roma, Roma, Italy
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia
Roma, Roma, Italy
Niigata National Hospital, National Hospital Organization
Kashiwazaki, Niigata, Japan
The University of Osaka Hospital
Suita-shi, Osaka, Japan
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, Japan
National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
Kodaira, Tokyo, Japan
Yamaguchi University Hospital
Ube-Shi, Yamaguchi, Japan
University of Maastricht
Maastricht, Netherlands
Radboud University Medical Center
Nijmegen, Netherlands
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
Northern Care Alliance NHS Foundation Trust
Salford, Lancashire, United Kingdom