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New drug DYNE-101 aims to ease muscle stiffness in rare disease

NCT ID NCT05481879

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

What the study statuses mean

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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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tests a new medicine, DYNE-101, in 116 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goals are to check if the drug is safe and if it can reduce muscle stiffness and improve muscle function. Participants receive the drug or a placebo by IV over several months, with long-term follow-up.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 116 people

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

Started

Sep 2022

Expected to finish

Jul 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

18 to 65 years

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 with trinucleotide repeat size \>100. * Age of onset of DM1 muscle symptoms ≥12 years. * Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator. * Hand grip strength and ankle dorsiflexion strength. * Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses. Exclusion Criteria: * History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study. * History of anaphylaxis. * Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments. * Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments. * Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator. * Percent predicted forced vital capacity (FVC) \<50%. * History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study. * Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide. * Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments. * Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers. Note: Other inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

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

Myotonic dystrophy type 1 (DM1)

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

    20 sites in 8 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

  • CHU de Nantes

    RECRUITING

    Nantes, 44093, France

  • Centro Clinico Nemo

    RECRUITING

    Milan, 20162, Italy

  • Charité - Universitätsmedizin Berlin

    RECRUITING

    Berlin, 10117, Germany

  • Fondazione Policlinico Universitario A Gemelli-Rome

    RECRUITING

    Rome, 00168, Italy

  • Indiana University School of Medicine

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Institut de Myologie

    RECRUITING

    Paris, 75013, France

  • John Walton Muscular Dystrophy Research Centre

    RECRUITING

    Newcastle upon Tyne, United Kingdom

  • Ludwig Maximilians University, Munich - Friedrich Baur Institut

    RECRUITING

    Munich, 80336, Germany

  • NZCR Auckland

    RECRUITING

    Auckland, 1023, New Zealand

  • Neurology Rare Disease Center

    RECRUITING

    Denton, Texas, 76208, United States

    Contact Email: •••••@•••••

  • Radboud Medical Center

    RECRUITING

    Nijmegen, Netherlands

    Contact Email: •••••@•••••

  • Salford Royal Hospital

    RECRUITING

    Salford, M6 8HD, United Kingdom

  • St. Vincent's Hospital

    RECRUITING

    Fitzroy, Victoria, 3065, Australia

  • Stanford University

    RECRUITING

    Stanford, California, 94305, United States

  • University College London Hospitals

    RECRUITING

    London, NW1 2BU, United Kingdom

  • University of Florida College of Medicine

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Rochester Medical Center

    RECRUITING

    Rochester, New York, 14642, United States

  • Virginia Commonwealth University (VCU)

    RECRUITING

    Richmond, Virginia, 23298, United States

  • Washington University in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

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