Experimental drug losmapimod tested for rare muscle disease

NCT ID NCT04264442

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

Summary

This phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminated early, so the full results are not available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Losmapimod (a drug taken as a pill twice daily)

What this could lead to

If it works, this could point toward a treatment that slows or controls muscle weakness in people with FSHD.

What could go wrong

This trial was terminated early, so results are limited. It is a small, phase 2 study, and the drug may not prove effective or safe in larger groups.

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.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1 muscular dystrophy musculoskeletal system disorder neuromuscular disease

As listed by the trial registrant

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

Contacts and locations

Locations

  • CHU de NICE- CHU pasteur2

    Nice, 06001, France

  • Hospital UiP La Fe

    Valencia, 46026, Spain

  • Hospital de la Sta Creu i St Pau

    Barcelona, 08041, Spain

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Montreal Neurological Institute and Hospital

    Montreal, Quebec, H3A 2B4, Canada

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Ottawa Hospital Research Institute

    Ottawa, Ontario, K1Y 4E9, Canada

  • University of California Irvine

    Irvine, California, 92868, United States

  • University of California Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01655, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • University of Washinton Medical Center

    Seattle, Washington, 98195, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States