Apitegromab for FSHD in Adults (Phase 2)

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled Phase 2 trial conducted at multiple centers to evaluate the efficacy, safety, and tolerability of apitegromab in people with facioscapulohumeral muscular dystrophy. Participants are assigned to receive either apitegromab or placebo, and outcomes include functional and safety assessments over a defined period. Eligible adults have a genetic diagnosis of FSHD, certain disease severity, and a baseline walking speed within a specified range. The study excludes individuals with prior anti-myostatin therapies, certain prior treatments, or hypersensitivities to monoclonal antibodies. Lead sponsorship is provided by Scholar Rock, Inc., with the National Neuromuscular Research Institute as the site.

Why it matters

Who can join: the full rules

Inclusion Criteria:

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

Exclusion Criteria:

1. 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
2. Treatment with other investigational drugs in a clinical trial within 3 months or 5 half-lives, whichever is longer, before screening
3. Previous treatment with apitegromab, or with other anti-myostatin therapies, including activin receptor antagonists
4. Current or prior use of anabolic steroids, growth hormones, glucagon-like peptide-1 receptor agonist or other substances with known effects on muscle.
5. 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
6. Use of systemic or corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
7. Pregnant or breastfeeding.
8. 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.
9. History of alcoholism, or illicit drug use (drugs that are illegal and have not been prescribed).
10. Taking medications that impede coagulation or platelet aggregation or has a history or active coagulopathy disorder.
11. 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 it's running

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

Study contacts

• Study coordinator: Scholar Rock, Inc. Clinical Trials Administration
• Email: MedicalInformation@scholarrock.com
• Phone: 857-259-3860

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• BetterLife FSHD: a patient-driven health platform

  This project tries to connect people with FSHD to personalized resources and collect their health experiences so they can be matched to relevant studies and help improve care.

  Recruiting FSHD Society · Randolph, Massachusetts · NCT07409142

• Del-brax (AOC 1020) intravenous treatment for people with FSHD

  This study will try regular IV infusions of del-brax (AOC 1020) in adults with FSHD to see if it slows muscle weakness and helps preserve walking ability.

  Recruiting at 28 sites Phase 3 Avidity Biosciences, Inc. · Orange, California and 45 other locations · NCT07038200

• Apitegromab for infants under 2 with spinal muscular atrophy

  This trial will test whether adding apitegromab to approved SMA therapy helps infants under 2 with 5q SMA reach better motor milestones.

  Recruiting at 23 sites Phase 2 Scholar Rock, Inc. · Phoenix, Arizona and 22 other locations · NCT07047144

• Personalized exercise treatment for adults with rare neuromuscular disorders

  This study is testing if a personalized exercise program can help adults with rare neuromuscular disorders improve their balance and physical function better than regular care.

  Recruiting at 2 sites Oslo University Hospital · Bergen and 4 other locations · NCT06708468

• Developing new biomarkers for muscular dystrophies

  This study is trying to find easier and less painful ways to measure how serious muscular dystrophies are by looking at urine and blood samples and using simple tests, to help improve diagnosis and treatment for people with these conditions.

  Recruiting at 4 sites Massachusetts General Hospital · Boston, Massachusetts and 4 other locations · NCT05019625

• Collecting motor and functional outcomes in patients with FSHD

  This study is tracking how Facioscapulohumeral Muscular Dystrophy affects movement and daily activities in about 450 people over three years to learn more about the condition.

  Recruiting at 18 sites University of Kansas Medical Center · Los Angeles, California and 20 other locations · NCT04635891

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.