Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Start Date
5/14/2026
Completion Date
1/1/2029
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).
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.
Eligibility Criteria
Age Range: 16 years to No maximum
Interventions
zeleciment basivarsen (DYNE-101)
Placebo
Conditions
Locations
UCSD - Altman Clinical and Translational Research Institute
La Jolla, California 92037
United States
Rare Disease Research, LLC
Atlanta, Georgia 30329
United States
IU Health Neuroscience Center
Indianapolis, Indiana 46202
United States
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri 65211
United States
Rare Disease Research, LLC
Hillsborough, North Carolina 27278
United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
United States
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah 84132
United States
Virginia Commonwealth University - Biotech One
Richmond, Virginia 23298
United States
UZ Leuven
Leuven, 3000
Belgium
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
Copenhagen, 2100
Denmark
CHU Marseille - Hôpital de la Timone (APHM)
Marseille, 13385
France
CHU de Nantes - Hôtel-Dieu
Nantes, 44000
France
Pitié-Salpêtrière Hospital
Paris, 75013
France
CHU de Toulouse - Hôpital Pierre-Paul Riquet
Toulouse, 31300
France
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
Munich, Bavaria 80336
Germany
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen
Bonn, North Rhine-Westphalia 53127
Germany
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
Berlin, State of Berlin 13125
Germany
Centro Clinico Nemo Milano, Fondazione Serena ETS
Milan, Milano 20162
Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile
Roma, Roma 00168
Italy
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia
Roma, Roma 00189
Italy
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka 560-8552
Japan
Yamaguchi University Hospital
Ube-Shi, Yamaguchi 755-8505
Japan
Hospital Universitario Infanta Sofía
San Sebastián de los Reyes, Madrid 28702
Spain
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London, England WC1N 3BG
United Kingdom
Northern Care Alliance NHS Foundation Trust
Salford, Lancashire M6 8HD
United Kingdom