Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

ConditionsFacioscapulohumeral Muscular Dystrophy FSHD

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18-60

SponsorScholar Rock, Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

Apitegromab
Drug
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.
Placebo
Drug
Placebo is administered every 4 weeks by intravenous (IV) infusion and does not contain the active ingredient.

Treatment groups

60 Participants

are divided into 2 treatment groups

Group A: ApitegromabExperimental treatment 1 intervention

Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

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

Time frame

52 Weeks

Secondary outcomes

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

Time frame

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

Time frame

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

Time frame

24 Weeks and 52 Weeks

Evaluate the pharmacokinetics of apitegromab in participants with FSHD

Serum concentrations of apitegromab

Time frame

52 Weeks

Other outcomes

Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

Change from baseline in quantitative muscle testing in kilogram-force. Higher score indicates greater function.

Time frame

52 Weeks

Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

Change from baseline in relative surface area as assessed by reachable workspace. Higher score indicates greater function.

Time frame

52 Weeks

Evaluate improvement(s) over time in motor function in participants with FSHD receiving apitegromab

Change from baseline in FSHD composite outcome measure (FSHD-COM) total score (0 - 72 scale; Higher score indicates greater impairment)

Time frame

52 Weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Scholar Rock, Inc.

Lead sponsor

Source ClinicalTrials.gov

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