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

Hoffmann-La Roche15 sites in 4 countries51 target enrollmentFebruary 7, 2023

ConditionsFacioscapulohumeral Muscular Dystrophy (FSHD)

InterventionsPlacebo RO7204239

DrugsPlacebo RO7204239

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Facioscapulohumeral Muscular Dystrophy (FSHD)
Sponsor: Hoffmann-La Roche
Enrollment: 51
Locations: 15
Primary Endpoint: Percentage of participants with adverse events (AEs)
Status: Active, not recruiting
Last Updated: 2 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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).

Registry

clinicaltrials.gov

Start Date

February 7, 2023

End Date

October 23, 2026

Last Updated

2 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Hoffmann-La Roche

Responsible Party

Sponsor

Eligibility Criteria

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)

Arms & Interventions

Placebo

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.

Intervention: Placebo

RO7204239

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.

Intervention: RO7204239

Outcomes

Primary Outcomes

Percentage of participants with adverse events (AEs)

Time Frame: Up to 2.5 years

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

Secondary Outcomes

Change from baseline in serum concentration of total latent myostatin(Through 2 years)
Change from baseline in serum concentration of mature myostatin(Through 2 years)
Percent change from baseline in CMV of 36 muscles based on whole body MRI(Weeks 28 and 52)
Percent change from baseline in CMV of quadriceps femoris muscles as assessed by MRI bilaterally(Week 28)
Change from baseline in fat fraction of quadriceps femoris muscles as assessed by MRI bilaterally(Weeks 28 and 52)
Percent change from baseline in CMV of tibialis anterior muscles as assessed by MRI bilaterally(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(Weeks 28 and 52)
Trough concentration (Ctrough) of RO7204239(Through 2 years)
Change from baseline in serum concentration of free latent myostatin(Through 2 years)
Change from baseline in fat fraction of 36 muscles based on whole body MRI(Weeks 28 and 52)
Change from baseline in fat fraction of tibialis anterior muscles as assessed by MRI bilaterally(Weeks 28 and 52)
Percent change from baseline in CMV of biceps brachii muscles as assessed by MRI bilaterally(Weeks 28 and 52)
Change from baseline in fat fraction of biceps brachii muscles as assessed by MRI bilaterally(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(Weeks 28 and 52)
Serum concentration of RO7204239(Through 2 years)
Percentage of participants with anti-drug antibodies (ADAs)(Baseline up to approximately 2 years)
Maximum serum concentration (Cmax) of RO7204239(Through 2 years)
Area under the concentration-time curve (AUC) of RO7204239(Through 2 years)