Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease

NCT ID NCT05479981

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

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

Summary

This study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infusion. Researchers are monitoring side effects and checking how the drug affects muscle tissue and disease markers.

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 slows or improves muscle function in people with DM1.
What could go wrong: This is a small, early-phase extension study with only 37 participants, so results may not apply to everyone. Long-term safety and effectiveness are still being evaluated.

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.

atrophic muscular disease hereditary disease inherited neurodegenerative disorder muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder myotonic dystrophy myotonic dystrophy type 1 myotonic syndrome nervous system disorder neurodegenerative disease neuromuscular disease

As listed by the trial registrant

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

DM1 GENETIC DISEASES, INBORN HEREDODEGENERATIVE DISORDERS, NERVOUS SYSTEM MUSCULAR DISEASES MUSCULAR DISORDERS, ATROPHIC MUSCULAR DYSTROPHIES MUSCULOSKELETAL DISEASES MYOTONIC DISORDERS MYOTONIC DYSTROPHY MYOTONIC DYSTROPHY 1 NERVOUS SYSTEM DISEASES NEURODEGENERATIVE DISEASES NEUROMUSCULAR DISEASES

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

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