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NCT07486934PHASE3Recruiting

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

Dyne Therapeutics

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

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.

Interventions

DRUG

zeleciment basivarsen (DYNE-101)

DRUG

Placebo

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Conditions

Myotonic Dystrophy Type 1 (DM1)DM1Myotonic DystrophySteinert DiseaseSteinert

Locations

UCSD - Altman Clinical and Translational Research Institute

La Jolla, California 92037

United States

UCLA Medical Center (Department of Neurology)

Los Angeles, California 90095

United States

Stanford Neuroscience Health Center

Palo Alto, California 94304

United States

UF Health: University of Florida Clinical Research Center

Gainesville, Florida 32610

United States

Rare Disease Research, LLC

Atlanta, Georgia 30329

United States

IU Health Neuroscience Center

Indianapolis, Indiana 46202

United States

Kennedy Krieger Institute

Baltimore, Maryland 21205

United States

Roy Blunt NextGen Precision Health Institute

Columbia, Missouri 65211

United States

University of Rochester Medical Center

Rochester, New York 14642

United States

Rare Disease Research, LLC

Hillsborough, North Carolina 27278

United States

Houston Methodist Neurological Institute

Houston, Texas 77030

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

St Vincent's Hospital, Melbourne

Fitzroy, Victoria 3065

Australia

UZA

Edegem, 2650

Belgium

UZ Leuven

Leuven, 3000

Belgium

Aarhus University Hospital, Department of Neurology

Aarhus, 8200

Denmark

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

Copenhagen, 2100

Denmark

AP-HP, Hôpital Raymond-Poincaré

Garches, 92380

France

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

Universitätsmedizin Göttingen

Göttingen, Lower Saxony 37075

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 Brescia, Fondazione Serena ETS

Gussago, Brescia 25064

Italy

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

Niigata National Hospital, National Hospital Organization

Kashiwazaki, Niigata 945-8585

Japan

The University of Osaka Hospital

Suita-shi, Osaka 565-0871

Japan

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-Shi, Osaka 560-8552

Japan

National Center Hospital, National Center of Neurology and Psychiatry (NCNP)

Kodaira, Tokyo 187-8551

Japan

Yamaguchi University Hospital

Ube-Shi, Yamaguchi 755-8505

Japan

University of Maastricht

Maastricht, 6229 HX

Netherlands

Radboud University Medical Center

Nijmegen, 6525 GA

Netherlands

Hospital Universitari Vall d'Hebron

Barcelona, Barcelona 08035

Spain

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