RecruitingPhase 3NCT07486934

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

Sponsor

Dyne Therapeutics

Enrollment

150 participants

Start Date

May 14, 2026

Study Type

INTERVENTIONAL

Conditions

Myotonic Dystrophy DM1 Steinert Disease Myotonic Dystrophy Type 1 (DM1)Steinert

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).

Eligibility

Min Age: 16 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called zeleciment basivarsen (DYNE-101) for people with dm1, myotonic dystrophy, and other related conditions. The study is currently recruiting participants at 5 locations. People eligible for this study include aged 16 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGzeleciment basivarsen (DYNE-101)

Administered by IV infusion

DRUGPlacebo

Administered by IV infusion

Locations(25)

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

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, United States

Virginia Commonwealth University - Biotech One

Richmond, Virginia, United States

UZ Leuven

Leuven, Belgium

Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)

Copenhagen, Denmark

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 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

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

Northern Care Alliance NHS Foundation Trust

Salford, Lancashire, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07486934

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