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

NCT ID NCT07486934

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 22 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 150 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Jan 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    37 sites in 11 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AP-HP, Hôpital Raymond-Poincaré

    RECRUITING

    Garches, 92380, France

  • Aarhus University Hospital, Department of Neurology

    RECRUITING

    Aarhus, 8200, Denmark

  • 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

  • Radboud University Medical Center

    RECRUITING

    Nijmegen, 6525 GA, Netherlands

    Contact Email: •••••@•••••

  • 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

  • St Vincent's Hospital, Melbourne

    RECRUITING

    Fitzroy, Victoria, 3065, Australia

  • Stanford Neuroscience Health Center

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Email: •••••@•••••

  • 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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Other studies related to the condition(s) this trial covers.