A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy

NCT05548556 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: BN43703
• Study Type: interventional
• Target: 51
• Locations: 4 countries
• Sites: 10

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).

Conditions

Facioscapulohumeral Muscular Dystrophy (FSHD)

Outcome Measures

Primary Outcomes (2)

• Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally

  Week 52

• Percentage of participants with adverse events (AEs)

  Up to 2.5 years

Secondary Outcomes (18)

• Change from baseline in serum concentration of total latent myostatin

  Through 2 years

• Change from baseline in serum concentration of free 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

• Change from baseline in fat fraction of 36 muscles based on whole body MRI

  Weeks 28 and 52

Study Arms (2)

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.

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.

Drug: RO7204239

Interventions

Placebo DRUG

Participants will receive subcutaneous (SC) placebo every 4 weeks (Q4W)

Placebo

RO7204239 DRUG

Participants will receive SC RO7204239 Q4W

RO7204239

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (10)

University of Irvine Medical Center (UCIMC)

Orange, California, 92868, United States

Location

Regents of the University of Colorado

Aurora, Colorado, 80045, United States

Location

University of Kansas Medical Center

Fairway, Kansas, 66205, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Virginia Commonwealth University Medical Center

Richmond, Virginia, 23298-0599, United States

Location

Rigshospitalet

København Ø, 2100, Denmark

Location

Policlinico Universitario Agostino Gemelli

Rome, Lazio, 00168, Italy

Location

Asst Grande Ospedale Metropolitano Niguarda

Milan, Lombardy, 20162, Italy

Location

National Hospital for Neurology and Neurosurgery,

London, WC1N 3BG, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2022
• First Posted: September 21, 2022
• Study Start: February 7, 2023
• Primary Completion: May 2, 2025
• Study Completion (Estimated): October 23, 2026
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (5); DK (1); IT (2); GB (2)