Facioscapulohumeral Muscular DystrophyPhase 2NCT07435129
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A Phase 2 study testing Apitegromab in people with Facioscapulohumeral Muscular Dystrophy and FSHD, run by Scholar Rock, Inc. at 1 site in United States.
Trial summary
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy, FSHD
- What is being tested?
- Apitegromab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2026to Dec 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Scholar Rock, Inc. Clinical Trials Administration contact
United States1 site, 1 recruiting
- Recruiting
National Neuromuscular Research Institute
Austin, Texas, 78759
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter, 52-Week Study Evaluating the Efficacy and Safety of Apitegromab in Participants With Facioscapulohumeral Muscular Dystrophy (FORGE)
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
- Facioscapulohumeral Muscular Dystrophy, FSHD
- Being tested
- Apitegromab, Placebo
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApitegromabDrug
Also called SRK-015
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.
PlaceboDrug
Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.
| Study group | Role | What they receive |
|---|---|---|
| Apitegromab | Experimental | 10 mg/kg IV Receives: Drug: Apitegromab |
| Placebo | Placebo comparator | Placebo IV Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 52 Weeks
Secondary outcomes (7)
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
Measured: 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
Measured: 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
Measured: 24 Weeks and 52 Weeks
Evaluate the pharmacokinetics of apitegromab in participants with FSHD
Serum concentrations of apitegromab
Measured: 52 Weeks
Evaluate the pharmacodynamics of apitegromab in participants with FSHD
Serum concentrations of total latent myostatin
Measured: 52 Weeks
Evaluate the safety and tolerability of apitegromab in participants with FSHD
Incidence of anti-drug antibodies against apitegromab in serum
Measured: 52 Weeks
Evaluate the safety and tolerability of apitegromab in FSHD participants
Incidence of adverse events and serious adverse events
Measured: 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start30 Jul 2026
- Primary completionJun 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facioscapulohumeral Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Scholar Rock, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07435129
- Sponsor's protocol ID
- SRK-015-009
- Status last verified
- Sep 2026
- First submitted
- 10 Feb 2026
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No