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New hope for FSHD: experimental drug AOC 1020 tested in humans

NCT ID NCT05747924

First seen Apr 29, 2026 · Last updated May 01, 2026 · Updated 1 time

Summary

This study tested an experimental drug called AOC 1020 in 90 adults with facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disease that causes progressive muscle weakness. The main goals were to check the drug's safety and how the body processes it. Participants received either the drug or a placebo by IV, and researchers also looked for early signs that the drug might help control the disease.

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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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Contacts and locations

Locations

  • Duke University

    Durham, North Carolina, 27708, United States

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • University College London

    London, WIT 7HA, United Kingdom

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of California San Diego

    San Diego, California, 92093, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Ottawa

    Ottawa, Ontario, K1Y 4E9, Canada

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Sheffield

    Sheffield, S10 2TN, United Kingdom

  • University of Texas Southwestern

    Dallas, Texas, 75390, United States

  • University of Washington

    Seattle, Washington, 98104, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

Conditions

Explore the condition pages connected to this study.