RO7204239 for Facioscapulohumeral Muscular Dystrophy

(MANOEUVRE Trial)

Not currently recruiting at 20 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

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. ^{1 2 3 4 5}

Why 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. ^{1 5 6 7 8}

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

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05548556

NCT05548556 | A Study to Evaluate RO7204239 in ...

The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal ...

2.

forpatients.roche.com

forpatients.roche.com/content/patient-platform/global/en/trials/muscle-and-peripheral-nerve-disease/facioscapulohumeral-muscular-dystrophy/a-study-to-evaluate-ro7204239-in-participants-with-faci-44329.pdf

A clinical trial to compare RO7204239 with placebo in people ...

The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11632561/

A 5‐year natural history cohort of patients with ...

In this study, we report the natural progression of FSHD and determine the feasibility of COAs for clinical trials.

4.

mdaconference.org

mdaconference.org/abstract-library/manoeuvre-study-design-a-study-of-gym329-ro7204239-in-patients-with-facioscapulohumeral-muscular-dystrophy-fshd/

A study of GYM329 (RO7204239) in patients with ...

This study will provide valuable information about the PD, safety, PK and efficacy of GYM329 treatment in patients with FSHD.

5.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12287641/

Current landscape for the management of ...

A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy (MANOEUVRE) 2024. [cited 2024 November 25] ...

6.

fshdsociety.org

fshdsociety.org/research-and-clinical-care/current-trials-and-studies/roche-manoeuvre-trial/

Roche's MANOEUVRE Trial

Test whether RO7204239 can help muscles grow; Evaluate how safe RO7204239 is and what the side effects of taking it might be. How does the medicine work? FSHD ...

7.

genentech-clinicaltrials.com

genentech-clinicaltrials.com/en/trials/muscle-and-peripheral-nerve-disease/facioscapulohumeral-muscular-dystrophy/a-study-to-evaluate-ro7204239-in-participants-with-faci-44329.html

Clinical trial for -Genentech A Member of the Roche Group

The purpose of this clinical trial is to compare the effects, good or bad, of RO7204239 against placebo in people with FSHD.

8.

clinicaltrials.eu

clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-ro7204239-for-patients-with-facioscapulohumeral-muscular-dystrophy/

Study on the Effects and Safety of RO7204239 for Patients ...

This clinical trial investigates the safety and effects of RO7204239, a humanised IgG1 monoclonal antibody, on patients suffering from the genetic muscle ...