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New drug AOC 1001 tested for rare muscle disease

NCT ID NCT05027269

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new drug called AOC 1001 in 39 adults with myotonic dystrophy type 1, a genetic muscle disorder. Participants received either the drug or a placebo by IV infusion. The goal was to check safety and how the drug moves through the body. Results will help decide if larger studies are warranted.

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
What this could lead to
If successful, this could point toward a treatment that controls or reduces symptoms of myotonic dystrophy type 1.
What could go wrong
This is an early phase 1/2 trial with only 39 participants, so results may not apply to everyone. The drug may cause side effects or fail to improve symptoms.

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.

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

Contacts and locations

Locations

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

More trials for these conditions

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