Experimental drug losmapimod tested for rare muscle disease
NCT ID NCT04264442
First seen Jun 27, 2026 ยท 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 โ 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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHU de NICE- CHU pasteur2
Nice, 06001, France
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Hospital UiP La Fe
Valencia, 46026, Spain
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Hospital de la Sta Creu i St Pau
Barcelona, 08041, Spain
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4, Canada
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Ohio State University
Columbus, Ohio, 43221, United States
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Ottawa Hospital Research Institute
Ottawa, Ontario, K1Y 4E9, Canada
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University of California Irvine
Irvine, California, 92868, United States
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University of California Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Utah
Salt Lake City, Utah, 84132, United States
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University of Washinton Medical Center
Seattle, Washington, 98195, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Inflammation may be key in rare muscle disease
- FSHD patients unite: new registry aims to accelerate research and care
- FSHD drug trial halted midway: what happened?
- New antibody aims to block muscle wasting in rare disease
- Could umbilical cord stem cells ease muscle weakness in FSHD?
- Experimental gene 'Silencer' therapy enters human testing for rare muscle disease