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Hope for muscle strength: new drug targets myotonic dystrophy

NCT ID NCT06411288

Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 02, 2026 · Updated 3 times

Summary

This phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the drug improves hand opening time and muscle strength.

What this could mean

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

Active substance
AOC 1001 (del-desiran)
What this could lead to
If successful, this could lead to the first approved treatment that improves hand function and muscle strength in people with myotonic dystrophy type 1.
What could go wrong
This is a late-stage trial, but the drug is still experimental. It may not work better than placebo, and there could be side effects from the intravenous infusion.

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

159 people

The number who actually took part.

Started

May 2024

Finished

Jul 2026

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 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.

Key Inclusion Criteria: * Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1 * Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening Key Exclusion Criteria: * Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling or unable to comply with contraceptive requirements * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Diabetes that is not adequately controlled * History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded. * Body Mass Index \> 35 kg/m2 at Screening * Recently treated with an investigational drug or biological agent * Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline. Note: Additional protocol defined Inclusion and Exclusion criteria 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 Dystrophy myotonic Myotonia Myotonic disorders Myotonic dystrophy Myotonic dystrophy 1 Myotonic dystrophy type 1 (DM1) Myotonic muscular dystrophy Steinert Steinert disease

Contacts and locations

Locations

  • AP-HP Hopital Pitie-Salpetriere

    Paris, 75013, France

  • Aarhus University Hospital

    Aarhus N, 8200, Denmark

  • Aomori Hospital

    Aomori, Aomori, 038-1331, Japan

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • Duke University Medical Center

    Durham, North Carolina, 27708, United States

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    Milan, 20162, Italy

  • Hospital Universitario Donostia

    Donostia / San Sebastian, 20014, Spain

  • Houston Methodist Neurological Institute

    Houston, Texas, 77030, United States

  • Indiana University (IU)

    Indianapolis, Indiana, 46202, United States

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    Munich, 80336, Germany

  • Maastricht University Medical Center

    Maastricht, 6229 HX, Netherlands

  • Montreal Neurological Institute

    Montreal, Quebec, H3A 2B4, Canada

  • National Center of Neurology and Psychiatry

    Tokyo, 187-8551, Japan

  • National Hospital Organization Osaka Toneyama Medical Center

    Osaka, 560-8552, Japan

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Osaka University Hospital

    Osaka, 565-0871, Japan

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • St. Georges University Hospitals NHS Foundation Trust

    London, SW17 0QT, United Kingdom

  • Stanford University

    Stanford, California, 94305, United States

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA, Netherlands

  • The Ottawa Hospital

    Ottawa, Ontario, K1Y 4E9, Canada

  • University College London Hospital

    London, NW1 2BU, United Kingdom

  • University Research Center of South Florida

    Tampa, Florida, 33612, United States

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio, 45219, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Washington

    Seattle, Washington, 98104, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Wake Forest

    Winston-Salem, North Carolina, 27157, United States

More trials for these conditions

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