Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
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
- Myotonic Dystrophy Type 1 (DM1)
- DM1
- Myotonic Dystrophy
- Steinert Disease
- Steinert
At a glance
- Phase
- Phase 3
- Study type
- Interventional
- Sponsor
- Dyne Therapeutics
- Enrolment target
- 150
- Started
- 14 May 2026
- Main results due
- July 2028
- Study sites
- 39
- Registry updated
- 25 September 2026
Can you take part?
- Aged 16 years and over.
- Open to any sex.
- You need the condition being studied — healthy volunteers are not accepted.
These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.
Read the full eligibility criteria
Inclusion Criteria: * Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable. * Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed). * Body mass index (BMI) less than (\<) 35 kilograms per meter square (kg/m\^2). Exclusion Criteria: * A known diagnosis of congenital DM1. * History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator. * Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments. Note: Other inclusion and exclusion criteria may apply.
What this study is about
In the sponsor’s own words, from the registry.
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).
Study sites(39)
UCSD - Altman Clinical and Translational Research Institute
La Jolla, California, United States
Recruiting
UCLA Medical Center (Department of Neurology)
Los Angeles, California, United States
Recruiting
Stanford Neuroscience Health Center
Palo Alto, California, United States
Recruiting
Rare Disease Research, LLC
Atlanta, Georgia, United States
Recruiting
IU Health Neuroscience Center
Indianapolis, Indiana, United States
Recruiting
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States
Recruiting
University of Rochester Medical Center
Rochester, New York, United States
Recruiting
Rare Disease Research, LLC
Hillsborough, North Carolina, United States
Recruiting
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Recruiting
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States
Recruiting
Virginia Commonwealth University - Biotech One
Richmond, Virginia, United States
Recruiting
St Vincent's Hospital, Melbourne
Fitzroy, Victoria, Australia
Recruiting
UZA
Edegem, Belgium
Recruiting
UZ Leuven
Leuven, Belgium
Recruiting
Aarhus University Hospital, Department of Neurology
Aarhus, Denmark
Recruiting
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
Copenhagen, Denmark
Recruiting
AP-HP, Hôpital Raymond-Poincaré
Garches, France
Recruiting
CHU Marseille - Hôpital de la Timone (APHM)
Marseille, France
Recruiting
CHU de Nantes - Hôtel-Dieu
Nantes, France
Recruiting
Pitié-Salpêtrière Hospital
Paris, France
Recruiting
CHU de Toulouse - Hôpital Pierre-Paul Riquet
Toulouse, France
Recruiting
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
Munich, Bavaria, Germany
Recruiting
Universitätsmedizin Göttingen
Göttingen, Lower Saxony, Germany
Recruiting
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen
Bonn, North Rhine-Westphalia, Germany
Recruiting
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
Berlin, State of Berlin, Germany
Recruiting
Centro Clinico Nemo Brescia, Fondazione Serena ETS
Gussago, Brescia, Italy
Recruiting
Centro Clinico Nemo Milano, Fondazione Serena ETS
Milan, Milano, Italy
Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile
Roma, Roma, Italy
Recruiting
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia
Roma, Roma, Italy
Recruiting
Niigata National Hospital, National Hospital Organization
Kashiwazaki, Niigata, Japan
Recruiting
The University of Osaka Hospital
Suita-shi, Osaka, Japan
Recruiting
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, Japan
Recruiting
National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
Kodaira, Tokyo, Japan
Recruiting
Yamaguchi University Hospital
Ube-Shi, Yamaguchi, Japan
Recruiting
University of Maastricht
Maastricht, Netherlands
Recruiting
Radboud University Medical Center
Nijmegen, Netherlands
Recruiting
Hospital Universitario Infanta Sofía
San Sebastián de los Reyes, Madrid, Spain
Recruiting
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London, England, United Kingdom
Recruiting
Northern Care Alliance NHS Foundation Trust
Salford, Lancashire, United Kingdom
Recruiting
Showing 39 of 39 sites. 39 of the 39 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.
Source: ClinicalTrials.gov record NCT07486934. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.
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