RO7204239 for Facioscapulohumeral Muscular Dystrophy (MANOEUVRE Trial)

Phase 2

Recruiting

Research Sponsored by Hoffmann-La Roche

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2.5 years

Summary

This trial will assess the safety and effectiveness of a drug to treat facioscapulohumeral muscular dystrophy (FSHD).

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include allergic reactions like anaphylaxis to RO7204239 or its components. Since it's an injection treatment, there might also be local skin reactions at the site of administration.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2.5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2.5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally

Percentage of participants with adverse events (AEs)

Secondary study objectives

Area under the concentration-time curve (AUC) of RO7204239

Change from baseline in fat fraction of 36 muscles based on whole body MRI

Change from baseline in fat fraction of biceps brachii muscles as assessed by MRI bilaterally

+15 more

Trial Design

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.

Do I qualify?Apply below to find out