Not Yet RecruitingPhase 2

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

60 participantsStarted 2026-08

Plain-language summary

A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD

Age range18 Years – 60 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Male or female participants, 18 to 60 years of age at the time of informed consent.
✓. Genetic diagnosis of FSHD Type 1 or FSHD Type 2, confirmed with the appropriate documentation from an accredited laboratory
✓. Clinical severity score of 1.5 to 3.0 (Ricci score; range 0 to 5), inclusive, at screening
✓. Baseline 10-meter walk/run test time ≤5 seconds

✕. Prior history of a hypersensitivity reaction to a mAb or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab
✕. Treatment with other investigational drugs in a clinical trial within 3 months or 5 half-lives, whichever is longer, before screening
✕. Previous treatment with apitegromab, or with other anti-myostatin therapies, including activin receptor antagonists
✕. Current or prior use of anabolic steroids, growth hormones, glucagon-like peptide-1 receptor agonist or other substances with known effects on muscle.
✕. Use of therapies with potentially significant muscle effects (eg, androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (eg, acetylcholinesterase inhibitors) within 60 days before screening
✕. Use of systemic or corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
✕. Pregnant or breastfeeding.
✕. Contraindications for MRI that may include, but are not limited to, certain implanted electronic devices, cochlear implants, metallic foreign bodies, vascular clips, and metallic implants; or claustrophobia, contrast agent allergies, inability to lie still, or external medical devices that may not be removed.

Assess the efficacy of apitegromab compared with placebo in participants with FSHD

Timeframe: 52 Weeks

NCT IDNCT07435129

SponsorScholar Rock, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-06

Scholar Rock, Inc. Clinical Trials Administration

Who can participate

Age range18 Years – 60 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Male or female participants, 18 to 60 years of age at the time of informed consent.
✓. Genetic diagnosis of FSHD Type 1 or FSHD Type 2, confirmed with the appropriate documentation from an accredited laboratory
✓. Clinical severity score of 1.5 to 3.0 (Ricci score; range 0 to 5), inclusive, at screening
✓. Baseline 10-meter walk/run test time ≤5 seconds

✕. Prior history of a hypersensitivity reaction to a mAb or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab
✕. Treatment with other investigational drugs in a clinical trial within 3 months or 5 half-lives, whichever is longer, before screening
✕. Previous treatment with apitegromab, or with other anti-myostatin therapies, including activin receptor antagonists
✕. Current or prior use of anabolic steroids, growth hormones, glucagon-like peptide-1 receptor agonist or other substances with known effects on muscle.
✕. Use of therapies with potentially significant muscle effects (eg, androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (eg, acetylcholinesterase inhibitors) within 60 days before screening
✕. Use of systemic or corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
✕. Pregnant or breastfeeding.
✕. Contraindications for MRI that may include, but are not limited to, certain implanted electronic devices, cochlear implants, metallic foreign bodies, vascular clips, and metallic implants; or claustrophobia, contrast agent allergies, inability to lie still, or external medical devices that may not be removed.