A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy (NCT05548556) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
United States51 participantsStarted 2023-02-07
Plain-language 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).
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
* 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 …
What they're measuring
1
Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally
Timeframe: Week 52
2
Percentage of participants with adverse events (AEs)