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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY 1 are added.
By submitting, you agree to our Terms of use
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
-
Radboud University Medical Center
Nijmegen, 9101, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New hope for FSHD: Long-Term drug safety trial underway
- New hope for FSHD: experimental drug AOC 1020 completes early testing
- FSHD patients unite: new registry aims to accelerate research and care
- Sound waves could spot early signs of rare muscle disease
- Hope for FSHD: new drug enters final stage of testing