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Not yet recruiting NCT07435129

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

Phase II Interventional Facioscapulohumeral Muscular Dystrophy FSHD

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Apitegromab, Placebo.
Who it may be relevant to
Registry conditions: Facioscapulohumeral Muscular Dystrophy, FSHD. Basic parameters: 18 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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

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)

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]
Secondary outcome measures (7)
  • Further assess the efficacy of apitegromab compared with placebo in participants with FSHD [Time frame: 24 Weeks]
  • Further assess the efficacy of apitegromab compared with placebo in participants with FSHD [Time frame: 24 Weeks and 52 Weeks]
  • Further assess the efficacy of apitegromab compared with placebo in participants with FSHD [Time frame: 24 Weeks and 52 Weeks]
  • Evaluate the pharmacokinetics of apitegromab in participants with FSHD [Time frame: 52 Weeks]
  • Evaluate the pharmacodynamics of apitegromab in participants with FSHD [Time frame: 52 Weeks]
  • Evaluate the safety and tolerability of apitegromab in participants with FSHD [Time frame: 52 Weeks]
  • Evaluate the safety and tolerability of apitegromab in FSHD participants [Time frame: 52 Weeks]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Triple blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07435129 · SRK-015-009

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗