RO7204239 for Facioscapulohumeral Muscular Dystrophy

(MANOEUVRE Trial)

Recruiting at 17 trial locations

Overseen By Reference Study ID Number: BN43703 https://forpatients.roche.com/

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Hoffmann-La Roche

Must not be taking: Anti-myostatin, Anabolic steroids

Disqualifiers: Pregnancy, Cardiovascular disease, Malignancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

48 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as oral salbutamol, creatine, growth hormones, anabolic steroids, and chronic oral or injected corticosteroids, at least 90 days before joining. Inhaled corticosteroids are allowed.

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).

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

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.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-treatment

Participants complete a 4-week pre-treatment period to collect baseline movement data with a wearable device

4 weeks

Treatment

Participants receive SC RO7204239 or placebo every 4 weeks for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with RO7204239 for an additional 52 weeks

52 weeks

Treatment Details

Interventions

• RO7204239

Trial Overview 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.

Participant Groups

Group I: RO7204239 Experimental Treatment 1 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: Placebo Placebo Group 1 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.

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University