Facioscapulohumeral Muscular Dystrophy (FSHD)Phase 2MANOEUVRENCT05548556
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
A Phase 2 study testing RO7204239 in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Hoffmann-La Roche at 9 sites in 4 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- What is being tested?
- RO7204239
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 51 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 7 Feb 2023to 9 Oct 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetic confirmation of FSHD1 or FSHD2
- Clinical findings consistent with FSHD
- Ability to walk unassisted
- Ricci Clinical Severity Scale score ≥ 2.5 and ≤ 4
- Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study
Who cannot join
- Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239
- Current or previous treatment (or receipt) of anti-myostatin therapies
- Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer
- Contraindications to MRI scans
- Presence of clinically significant ECG abnormalities
- Presence of clinically significant cardiovascular disease
Show all 15Show fewer
- Presence of clinically significant abnormal findings in echocardiography at screening, with the exception of mitral valve prolapse, which does not exclude participants from the study
- Any major illness within 1 month before screening
- Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7204239, or to the constituents of its formulation
- History of malignancy (except in situ basal cell carcinoma of the skin and in situ carcinoma of the cervix of the uterus that have been excised and resolved with documented clean margins on pathology)
- Any clinically relevant history of anaphylactic reaction requiring inotropic support
- Any abnormal skin conditions, pigmentation, or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection-site reactions to RO7204239
- Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening or longer, if judged by the investigator that it may affect motor function assessment
- Any planned surgery that may affect a participant's motor function assessment, including participants who have had surgery of scapular fixation within the 12 months preceding screening or that are planned during the study
- Use of the following medications within 90 days prior to enrollment: salbutamol or another β2-adrenergic agonist taken orally; creatine; recombinant human growth hormone; recombinant human insulin growth factor-1; testosterone, oxandrolone, or other anabolic steroid; chronic oral or parenteral use of corticosteroids (inhaled corticosteroid use is allowed) unless required to manage injection reactions;agents anticipated to increase or decrease muscle volume or strength
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
United States4 sites
Regents of the University of Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Kansas Medical Center
Fairway, Kansas, 66205
University of Irvine Medical Center (UCIMC)
Orange, California, 92868
Italy2 sites
Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162
Policlinico Universitario Agostino Gemelli
Rome, Lazio, 00168
United Kingdom2 sites
National Hospital for Neurology and Neurosurgery,
London, WC1N 3BG
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
About this study
Official title: A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
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).
- Being tested
- Placebo, RO7204239
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Participants will receive subcutaneous (SC) placebo every 4 weeks (Q4W)
RO7204239Drug
Participants will receive SC RO7204239 Q4W
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo |
| RO7204239 | Experimental | 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. Receives: Drug: RO7204239 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally
Measured: Week 52
Percentage of participants with adverse events (AEs)
Measured: Up to 2.5 years
Secondary outcomes (18)
Change from baseline in serum concentration of total latent myostatin
Measured: Through 2 years
Change from baseline in serum concentration of free latent myostatin
Measured: Through 2 years
Change from baseline in serum concentration of mature myostatin
Measured: Through 2 years
Percent change from baseline in CMV of 36 muscles based on whole body MRI
Measured: Weeks 28 and 52
Change from baseline in fat fraction of 36 muscles based on whole body MRI
Measured: Weeks 28 and 52
Percent change from baseline in CMV of quadriceps femoris muscles as assessed by MRI bilaterally
Measured: Week 28
Change from baseline in fat fraction of quadriceps femoris muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Percent change from baseline in CMV of tibialis anterior muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Change from baseline in fat fraction of tibialis anterior muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Percent change from baseline in CMV of biceps brachii muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Change from baseline in fat fraction of biceps brachii muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Percent change from baseline in contractile cross-sectional area (CSA) of skeletal muscle in the proximal lower limb muscles as assessed by MRI bilaterally
Measured: Weeks 28 and 52
Change from baseline in fat fraction of proximal lower limb muscles as assessed at a single mid-femur slice bilaterally by MRI
Measured: Weeks 28 and 52
Serum concentration of RO7204239
Measured: Through 2 years
Maximum serum concentration (Cmax) of RO7204239
Measured: Through 2 years
Area under the concentration-time curve (AUC) of RO7204239
Measured: Through 2 years
Trough concentration (Ctrough) of RO7204239
Measured: Through 2 years
Percentage of participants with anti-drug antibodies (ADAs)
Measured: Baseline up to approximately 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2022
- Study start7 Feb 2023
- Primary completion2 May 2025
- Study completion9 Oct 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facioscapulohumeral Muscular Dystrophy (FSHD)Studies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05548556
- Sponsor's protocol ID
- BN43703
- Acronym
- MANOEUVRE
- Status last verified
- Oct 2026
- First submitted
- 16 Sep 2022
- Last updated
- 15 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05548556
- Acronym / sponsor ID
- MANOEUVRE / BN43703
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 51 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 7 Feb 2023 (actual)
- Primary completion
- 2 May 2025 (actual)
- Study completion
- 9 Oct 2026 (actual)
- First posted
- 21 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboParticipants 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. |
| RO7204239 | Experimental | Drug: RO7204239Participants 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Genetic confirmation of FSHD1 or FSHD2
- Clinical findings consistent with FSHD
- Ability to walk unassisted
- Ricci Clinical Severity Scale score ≥ 2.5 and ≤ 4
- Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study
Key exclusion criteria (15 listed)
- Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239
- Current or previous treatment (or receipt) of anti-myostatin therapies
- Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer
- Contraindications to MRI scans
- Presence of clinically significant ECG abnormalities
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboParticipants will receive subcutaneous (SC) placebo every 4 weeks (Q4W) | Drug | Placebo |
| RO7204239Participants will receive SC RO7204239 Q4W | Drug | RO7204239 |
Primary outcome
Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally
Time frame: Week 52
Percentage of participants with adverse events (AEs)
Time frame: Up to 2.5 years
18 secondary outcomes
Change from baseline in serum concentration of total latent myostatin
Time frame: Through 2 years
Change from baseline in serum concentration of free latent myostatin
Time frame: Through 2 years
Change from baseline in serum concentration of mature myostatin
Time frame: Through 2 years
Percent change from baseline in CMV of 36 muscles based on whole body MRI
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of 36 muscles based on whole body MRI
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of quadriceps femoris muscles as assessed by MRI bilaterally
Time frame: Week 28
Change from baseline in fat fraction of quadriceps femoris muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of tibialis anterior muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of tibialis anterior muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of biceps brachii muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of biceps brachii muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in contractile cross-sectional area (CSA) of skeletal muscle in the proximal lower limb muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of proximal lower limb muscles as assessed at a single mid-femur slice bilaterally by MRI
Time frame: Weeks 28 and 52
Serum concentration of RO7204239
Time frame: Through 2 years
Maximum serum concentration (Cmax) of RO7204239
Time frame: Through 2 years
Area under the concentration-time curve (AUC) of RO7204239
Time frame: Through 2 years
Trough concentration (Ctrough) of RO7204239
Time frame: Through 2 years
Percentage of participants with anti-drug antibodies (ADAs)
Time frame: Baseline up to approximately 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
- Individual participant dataSharing planned: Yes. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
Related trials for Facioscapulohumeral Muscular Dystrophy (FSHD)
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
Facioscapulohumeral Muscular Dystrophy (FSHD)
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
RecruitingPhase 1
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD - Facioscapulohumeral Muscular Dystrophy
Not yet recruitingPhase 2
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular Dystrophy (FSHD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RO7204239 in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Hoffmann-La Roche at 9 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Italy, United Kingdom, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally (time frame: Week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.