RecruitingNCT07435129Updated Sep 2, 2026

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

A Phase 2 interventional study of Apitegromab and Placebo in Facioscapulohumeral Muscular Dystrophy and FSHD, sponsored by Scholar Rock, Inc.. Recruiting at 1 site in United States. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-09-02.

Sponsored by Scholar Rock, Inc. · Phase 2, Interventional, and Treatment

Phase: Phase 2
Study type: Interventional
Enrollment: 60
Allocation: Randomized
Ages: 18 Years to 60 Years
Sex: All

Study summary

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

Read the detailed description

This Phase 2, randomized, double-blind, placebo-controlled, multicenter study is designed to evaluate the efficacy, safety, and tolerability of apitegromab in participants with facioscapulohumeral muscular dystrophy (FSHD)

Conditions studied

Facioscapulohumeral Muscular Dystrophy
FSHD

Browse trials for

Muscular Dystrophy, Facioscapulohumeral 74

Who can participate

Ages eligible: 18 Years to 60 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

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.
History of alcoholism, or illicit drug use (drugs that are illegal and have not been prescribed).
Taking medications that impede coagulation or platelet aggregation or has a history or active coagulopathy disorder.
Any acute or comorbid condition interfering with the well-being of the participant within 7 days prior to screening, including active systemic infection, the need for acute treatment, or inpatient observation due to any reason

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Triple (Participant, Care provider, Outcomes assessor)
Enrollment: 60 participants (estimated)

Study arms

Experimental
Apitegromab
10 mg/kg IV
Drug: Apitegromab
Placebo comparator
Placebo
Placebo IV
Drug: Placebo

Interventions

DrugApitegromab
Apitegromab (SRK-015) is a fully human anti-promyostatin monoclonal antibody (mAb) that specifically binds to human pro/latent myostatin, inhibiting myostatin activation. Apitegromab will be administered every 4 weeks by intravenous (IV) infusion.
Also known as: SRK-015
DrugPlacebo
Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.

What researchers measure

Primary outcomes

Assess the efficacy of apitegromab compared with placebo in participants with FSHD
Percent change from baseline in total lean muscle volume (LMV) as measured by full body magnetic resonance imaging (MRI) at week 52
Time frame: 52 Weeks

Secondary outcomes

Further assess the efficacy of apitegromab compared with placebo in participants with FSHD
Percent change from baseline in total LMV as measured by full body MRI at week 24
Time frame: 24 Weeks
Further assess the efficacy of apitegromab compared with placebo in participants with FSHD
Change from baseline in additional muscle parameters, such as muscle fat fraction at week 24 and week 52
Time frame: 24 Weeks and 52 Weeks
Further assess the efficacy of apitegromab compared with placebo in participants with FSHD
Change from baseline in additional muscle parameters, such as muscle fat infiltration, at week 24 and week 52
Time frame: 24 Weeks and 52 Weeks
Evaluate the pharmacokinetics of apitegromab in participants with FSHD
Serum concentrations of apitegromab
Time frame: 52 Weeks
Evaluate the pharmacodynamics of apitegromab in participants with FSHD
Serum concentrations of total latent myostatin
Time frame: 52 Weeks
Evaluate the safety and tolerability of apitegromab in participants with FSHD
Incidence of anti-drug antibodies against apitegromab in serum
Time frame: 52 Weeks
Evaluate the safety and tolerability of apitegromab in FSHD participants
Incidence of adverse events and serious adverse events
Time frame: 52 Weeks

Other outcomes

Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab
Change from baseline in quantitative muscle testing in kilogram-force. Higher score indicates greater function.
Time frame: 52 Weeks
Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab
Change from baseline in relative surface area as assessed by reachable workspace. Higher score indicates greater function.
Time frame: 52 Weeks
Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab
Change from baseline in FSHD composite outcome measure (FSHD-COM) total score (0 - 72 scale; Higher score indicates greater impairment)
Time frame: 52 Weeks

Study locations

1 of 1 sites recruiting

National Neuromuscular Research Institute
Austin, Texas 78759, United States
Recruiting

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

Registry details

Key details

Study ID: NCT07435129
Lead sponsor: Scholar Rock, Inc.
Responsible party: Sponsor
First posted: Feb 27, 2026
Start date: Jul 30, 2026
Primary completion: Jun 2028 (estimated)
Completion: Dec 2028 (estimated)
Last update: Sep 2, 2026

Study contacts

Scholar Rock, Inc. Clinical Trials Administration

Contact

MedicalInformation@scholarrock.com

857-259-3860

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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