Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

NCT07435129 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: SRK-015-009
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Facioscapulohumeral Muscular Dystrophy; FSHD

Outcome Measures

Primary Outcomes (1)

• 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

  52 Weeks

Secondary Outcomes (7)

• Further assess the efficacy of apitegromab compared with placebo in participants with FSHD

  24 Weeks

• Further assess the efficacy of apitegromab compared with placebo in participants with FSHD

  24 Weeks and 52 Weeks

• Further assess the efficacy of apitegromab compared with placebo in participants with FSHD

  24 Weeks and 52 Weeks

• Evaluate the pharmacokinetics of apitegromab in participants with FSHD

  52 Weeks

• Evaluate the pharmacodynamics of apitegromab in participants with FSHD

  52 Weeks

Other Outcomes (3)

• Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

  52 Weeks

• Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

  52 Weeks

• Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

  52 Weeks

Study Arms (2)

Apitegromab

EXPERIMENTAL

10 mg/kg IV

Drug: Apitegromab

Placebo

PLACEBO COMPARATOR

Placebo IV

Drug: Placebo

Interventions

Apitegromab DRUG

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

Apitegromab

Placebo DRUG

Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Scholar Rock, Inc.: lead

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral

Interventions

apitegromab

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Scholar Rock, Inc. Clinical Trials Administration

CONTACT

857-259-3860; MedicalInformation@scholarrock.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2026
• First Posted: February 27, 2026
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share