New antibody aims to block muscle wasting in rare disease
NCT ID NCT05548556
First seen Nov 01, 2025 · Last updated Jun 19, 2026 · Updated 32 times
Summary
This Phase 2 trial tests a drug called RO7204239 in 51 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. The drug is an antibody that blocks myostatin, a protein that normally limits muscle growth, to help preserve or increase muscle. Participants receive either the drug or a placebo by injection every 4 weeks, and researchers measure changes in muscle volume and safety over time.
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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.
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
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Locations
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Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162, Italy
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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National Hospital for Neurology and Neurosurgery,
London, WC1N 3BG, United Kingdom
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Policlinico Universitario Agostino Gemelli
Rome, Lazio, 00168, Italy
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Regents of the University of Colorado
Aurora, Colorado, 80045, United States
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Rigshospitalet
København Ø, 2100, Denmark
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Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP, United Kingdom
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University of Irvine Medical Center (UCIMC)
Orange, California, 92868, United States
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University of Kansas Medical Center
Fairway, Kansas, 66205, United States
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Virginia Commonwealth University Medical Center
Richmond, Virginia, 23298-0599, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RO7204239 (a lab-made antibody that blocks myostatin, a protein that limits muscle growth)
What this could lead to
If it works, this could point toward a treatment that slows muscle loss and preserves strength in people with FSHD.
What could go wrong
This is a small, early Phase 2 trial with only 51 participants, so results may not apply to everyone. The drug may not improve muscle function or could cause side effects.
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.