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)
Scholar Rock, Inc.
Summary
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
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)
Eligibility
- Age range
- 18–60 years
- Sex
- All
- Healthy volunteers
- No
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. Treatmen…
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.
- DrugPlacebo
Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.
Location
- National Neuromuscular Research InstituteAustin, Texas