RO7204239 for Facioscapulohumeral Muscular Dystrophy
(MANOEUVRE Trial)

Recruiting in Palo Alto (17 mi)

+17 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Hoffmann-La Roche

Prior Safety Data

Trial Summary

What is the purpose of this trial?The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult participants with facioscapulohumeral muscular dystrophy (FSHD).

Eligibility Criteria

Adults who can walk on their own, have genetically confirmed FSHD1 or FSHD2 with specific clinical severity scores, and agree to keep their physical therapy routine stable during the study. Not for those pregnant, breastfeeding, hypersensitive to RO7204239 or its ingredients, with recent major illness/surgery affecting motor function, history of certain heart issues or malignancies.

Inclusion Criteria

I can walk by myself without help.

I agree to keep my physical therapy and exercise routines the same during the study.

My condition is genetically confirmed as FSHD1 or FSHD2.

+2 more

Exclusion Criteria

Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7204239, or to the constituents of its formulation

Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239

Contraindications to MRI scans

+12 more

Participant Groups

The trial is testing RO7204239—a monoclonal antibody targeting latent myostatin—against a placebo in adults with facioscapulohumeral muscular dystrophy (FSHD). It aims to assess the drug's effects on muscle function along with its safety and tolerability.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: RO7204239Experimental Treatment1 Intervention

Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive SC RO7204239 every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive subcutaneous (SC) placebo every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks.