NCT07435129 · Scholar Rock, Inc.
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
What this study is about
A randomly assigned Phase 2 study to evaluate the effectiveness and safety of apitegromab as a treatment given alone in participant with FSHD
View original scientific description
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
Interventions
DRUG
Apitegromab
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.
DRUG
Placebo
Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.
Primary outcome measures
Assess the efficacy of apitegromab compared with placebo in participants with FSHD
Time frame: 52 Weeks
Percent change from baseline in total lean muscle volume (LMV) as measured by full body magnetic resonance imaging (MRI) at week 52
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- 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
Where
- Austin, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 11, 2026 · Source of record for eligibility and locations