Extended trial tests promising drug for genetic muscle disease

NCT ID NCT05479981

Summary

This study continued testing an experimental drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. It followed 37 participants from a previous trial to check long-term safety and see if the drug continues to work when given every 8 weeks for up to 3 years. The main goal was to monitor side effects while also measuring how the drug affects the underlying genetic problem in muscle tissue.

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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

Conditions

Explore the condition pages connected to this study.