Experimental drug losmapimod tested in rare muscle disease – early hopes, but trial cut short
NCT ID NCT04004000
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was terminated early, so results are limited and it's too soon to know if the drug works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Losmapimod (15 mg twice daily by mouth)
What this could lead to
If successful, this could point toward a treatment that slows or manages the progression of FSHD1, a rare muscle-wasting disease.
What could go wrong
This was a very small, early pilot study (14 people) that was terminated early, so results are limited. The drug may not show meaningful benefit or may have side effects.
Disclaimer
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the original study
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Radboud University Medical Center
Nijmegen, 9101, Netherlands