A Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants with Facioscapulohumeral Muscular Dystrophy

Phase 1

• Conditions: Facioscapulohumeral muscular dystrophy (FSHD); MedDRA version: 20.0Level: PTClassification code: 10064087Term: Facioscapulohumeral muscular dystrophy Class: 100000004850; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: CTIS2022-503117-36-00
• Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-21
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

1. Age = 18 and = 65 years at the time of signing the Informed Consent Form, 2. Genetic confirmation of FSHD1 or FSHD2, 3. Clinical findings consistent with FSHD according to the investigator’s clinical judgment, 4. Ability to walk unassisted 10 meters (Timed 10-Meter Walk Test [10MWT]), 5. Ricci Clinical Severity Scale score = 2.5 and = 4, 6. Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study

Exclusion Criteria

1. Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239, 2. Current or previous treatment (or receipt) of anti-myostatin therapies, 3. Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer, 4. Contraindications to MRI scans, difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI, 5. Presence of clinically significant ECG abnormalities from average of triplicate measurement at screening indicating a safety risk for participants, 6. Presence of clinically significant cardiovascular disease indicating a safety risk for participants. •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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified