RO7204239 for Facioscapulohumeral Muscular Dystrophy
(MANOEUVRE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called RO7204239 for individuals with facioscapulohumeral muscular dystrophy (FSHD), a condition that causes muscle weakness. Researchers aim to determine the treatment's safety, how the body processes it, and its effect on muscle function. Participants will receive either the actual treatment or a placebo every four weeks for a year, with the option to continue the treatment afterward. This trial may suit those who can walk unaided and have been genetically confirmed to have FSHD. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.
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.
Is there any evidence suggesting that RO7204239 is likely to be safe for humans?
Research has shown that RO7204239 has been studied to understand its safety in humans. In earlier studies, researchers tested RO7204239, a humanized monoclonal antibody, to assess its tolerability and potential side effects. These studies suggest that it is generally well-tolerated.
Common side effects include mild reactions at the injection site. No serious side effects have been consistently linked to the treatment in the shared data. Since this study is in an early phase, researchers continue to collect safety information. However, reaching this phase indicates some confidence in its safety. While more data is needed, the early findings are promising for those considering joining the trial.12345Why do researchers think this study treatment might be promising for FSHD?
Unlike the standard treatments for Facioscapulohumeral Muscular Dystrophy, which often focus on symptom management through physical therapy and pain relief, RO7204239 introduces a new approach. It is administered subcutaneously and offers a targeted mechanism that researchers believe can address the disease at its core rather than just alleviating symptoms. Additionally, its unique delivery system, given every four weeks, might improve patient compliance and convenience compared to more frequent treatments. Researchers are eager to see if RO7204239 can provide a more effective and convenient alternative for patients with this condition.
What evidence suggests that RO7204239 might be an effective treatment for facioscapulohumeral muscular dystrophy?
Research has shown that RO7204239 is a specially designed antibody targeting a protein called myostatin, which usually limits muscle growth. By blocking myostatin, RO7204239 might help muscles grow stronger and larger, which is crucial for conditions like facioscapulohumeral muscular dystrophy (FSHD) that cause muscle weakness. Early results suggest this method could be effective, as it aims to increase muscle size and strength, vital for people with FSHD. Although more research is needed, this treatment appears promising for addressing the muscle weakness seen in FSHD. Participants in this trial will receive either RO7204239 or a placebo to evaluate its effectiveness.15678
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-treatment
Participants complete a 4-week pre-treatment period to collect baseline movement data with a wearable device
Treatment
Participants receive SC RO7204239 or placebo every 4 weeks for 52 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment with RO7204239 for an additional 52 weeks
What Are the Treatments Tested in This Trial?
Interventions
- RO7204239
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
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