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New drug aims to build muscle in rare muscular dystrophy

NCT ID NCT07435129

Recruiting now Disease control Sponsor: Scholar Rock, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 12, 2026 · Updated 3 times

Summary

This Phase 2 study tests a drug called apitegromab in 60 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. Participants will receive either the drug or a placebo every 4 weeks for a year. The main goal is to see if the drug increases total lean muscle volume, with a secondary focus on muscle function and safety.

What this could mean

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

Active substance
apitegromab (a lab-made antibody that blocks a protein called myostatin, which limits muscle growth)
What this could lead to
If it works, this could point toward a treatment that helps people with FSHD build and maintain muscle, slowing the disease's progression.
What could go wrong
This is an early Phase 2 trial with only 60 people, so results may not apply to everyone. The main goal is muscle volume, not yet proven to improve daily life. Side effects from the IV infusions are possible.

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.

facioscapulohumeral muscular dystrophy

As listed by the trial registrant

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

FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY FSHD

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • National Neuromuscular Research Institute

    RECRUITING

    Austin, Texas, 78759, United States

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