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New drug DYNE-101 aims to ease muscle symptoms in DM1

NCT ID NCT07486934

Recruiting now Disease control Sponsor: Dyne Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 17 times

Summary

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a chair, walk, and open their hands, along with overall health and safety.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
zeleciment basivarsen (DYNE-101)
What this could lead to
If successful, this could lead to a treatment that improves muscle function and daily living for people with myotonic dystrophy type 1.
What could go wrong
This is a Phase 3 trial, but it is still experimental. The drug may not work better than placebo, and there could be side effects from the intravenous infusions.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Myotonia myotonic dystrophy myotonic dystrophy type 1 myotonic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DM1 MYOTONIC DYSTROPHY MYOTONIC DYSTROPHY TYPE 1 (DM1) STEINERT STEINERT DISEASE

Contacts and locations

Locations

  • AP-HP, Hôpital Raymond-Poincaré

    RECRUITING

    Garches, 92380, France

  • Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia

    RECRUITING

    Roma, Roma, 00189, Italy

  • CHU Marseille - Hôpital de la Timone (APHM)

    RECRUITING

    Marseille, 13385, France

  • CHU de Nantes - Hôtel-Dieu

    RECRUITING

    Nantes, 44000, France

  • CHU de Toulouse - Hôpital Pierre-Paul Riquet

    RECRUITING

    Toulouse, 31300, France

  • Centro Clinico Nemo Brescia, Fondazione Serena ETS

    RECRUITING

    Gussago, Brescia, 25064, Italy

    Contact Email: •••••@•••••

  • Centro Clinico Nemo Milano, Fondazione Serena ETS

    RECRUITING

    Milan, Milano, 20162, Italy

  • Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit

    RECRUITING

    Berlin, State of Berlin, 13125, Germany

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile

    RECRUITING

    Roma, Roma, 00168, Italy

  • Hospital Universitario Infanta Sofía

    RECRUITING

    San Sebastián de los Reyes, Madrid, 28702, Spain

  • IU Health Neuroscience Center

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt

    RECRUITING

    Munich, Bavaria, 80336, Germany

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

    RECRUITING

    Kodaira, Tokyo, 187-8551, Japan

  • National Hospital Organization Osaka Toneyama Medical Center

    RECRUITING

    Toyonaka-Shi, Osaka, 560-8552, Japan

  • Niigata National Hospital, National Hospital Organization

    RECRUITING

    Kashiwazaki, Niigata, 945-8585, Japan

  • Northern Care Alliance NHS Foundation Trust

    RECRUITING

    Salford, Lancashire, M6 8HD, United Kingdom

  • Pitié-Salpêtrière Hospital

    RECRUITING

    Paris, 75013, France

  • Rare Disease Research, LLC

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Rare Disease Research, LLC

    RECRUITING

    Hillsborough, North Carolina, 27278, United States

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

    RECRUITING

    Copenhagen, 2100, Denmark

  • Roy Blunt NextGen Precision Health Institute

    RECRUITING

    Columbia, Missouri, 65211, United States

  • The University of Osaka Hospital

    RECRUITING

    Suita-shi, Osaka, 565-0871, Japan

  • The University of Texas Health Science Center at San Antonio

    RECRUITING

    San Antonio, Texas, 78229, United States

  • UCLA Medical Center (Department of Neurology)

    RECRUITING

    Los Angeles, California, 90095, United States

  • UCSD - Altman Clinical and Translational Research Institute

    RECRUITING

    La Jolla, California, 92037, United States

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • UZA

    RECRUITING

    Edegem, 2650, Belgium

  • University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery

    RECRUITING

    London, England, WC1N 3BG, United Kingdom

  • University of Maastricht

    RECRUITING

    Maastricht, 6229 HX, Netherlands

  • University of Utah Clinical Neurosciences Center

    RECRUITING

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen

    RECRUITING

    Bonn, North Rhine-Westphalia, 53127, Germany

  • Virginia Commonwealth University - Biotech One

    RECRUITING

    Richmond, Virginia, 23298, United States

  • Yamaguchi University Hospital

    RECRUITING

    Ube-Shi, Yamaguchi, 755-8505, Japan

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