Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease, Steinert

In brief

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).

Key facts

Study ID

NCT07486934

Run by

Dyne Therapeutics

People needed

150

Starts

2026-05-14

Expected to finish

2029-01-01

Last updated by the study team

2026-07-31

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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.

Where it is running

• UCSD - Altman Clinical and Translational Research Institute — La Jolla, California, United States (enrolling)
• Rare Disease Research, LLC — Atlanta, Georgia, United States (enrolling)
• IU Health Neuroscience Center — Indianapolis, Indiana, United States (enrolling)
• Roy Blunt NextGen Precision Health Institute — Columbia, Missouri, United States (enrolling)
• Rare Disease Research, LLC — Hillsborough, North Carolina, United States (enrolling)
• The University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States (enrolling)
• University of Utah Clinical Neurosciences Center — Salt Lake City, Utah, United States (enrolling)
• Virginia Commonwealth University - Biotech One — Richmond, Virginia, United States (enrolling)
• UZ Leuven — Leuven, Belgium (enrolling)
• Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077) — Copenhagen, Denmark (enrolling)
• CHU Marseille - Hôpital de la Timone (APHM) — Marseille, France (enrolling)
• CHU de Nantes - Hôtel-Dieu — Nantes, France (enrolling)
• Pitié-Salpêtrière Hospital — Paris, France (enrolling)
• CHU de Toulouse - Hôpital Pierre-Paul Riquet — Toulouse, France (enrolling)
• LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt — Munich, Bavaria, Germany (enrolling)
• Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen — Bonn, North Rhine-Westphalia, Germany (enrolling)
• Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit — Berlin, State of Berlin, Germany (enrolling)
• Centro Clinico Nemo Milano, Fondazione Serena ETS — Milan, Milano, Italy (enrolling)
• Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile — Roma, Roma, Italy (enrolling)
• Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia — Roma, Roma, Italy (enrolling)
• National Hospital Organization Osaka Toneyama Medical Center — Toyonaka-Shi, Osaka, Japan (enrolling)
• Yamaguchi University Hospital — Ube-Shi, Yamaguchi, Japan (enrolling)
• Hospital Universitario Infanta Sofía — San Sebastián de los Reyes, Madrid, Spain (enrolling)
• University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery — London, England, United Kingdom (enrolling)
• Northern Care Alliance NHS Foundation Trust — Salford, Lancashire, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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