New hope for DM1: Long-Term trial of AOC 1001 underway

NCT ID NCT07008469

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will receive the drug by IV infusion. Researchers will monitor side effects and how well the drug works over time.

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 confirm AOC 1001 as a long-term treatment option to manage symptoms and slow progression of myotonic dystrophy type 1.

What could go wrong

This is an open-label extension study, so there is no placebo group for comparison. Long-term safety and effectiveness are still being evaluated, and results may not apply to all patients.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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 MYOTONIA MYOTONIC DISORDERS MYOTONIC DYSTROPHY MYOTONIC DYSTROPHY 1 MYOTONIC DYSTROPHY TYPE 1 STEINERT 39 S DISEASE STEINERT DISEASE STEINERT MYOTONIC DYSTROPHY

Contacts and locations

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Locations

AP-HP Hopital Pitie-Salpetriere

Paris, 75013, France
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
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

München, 80336, Germany
Montreal Neurological Institute

Montreal, Quebec, H3A 2B4, Canada
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
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 Research Center of South Florida

Tampa, Florida, 33612, United States
University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States
University of Colorado

Denver, Colorado, 80045, United States
University of Florida

Gainesville, Florida, 32608, 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