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 - BN43703

F. Hoffmann-La Roche AG0 sites48 target enrollmentDecember 21, 2023

Overview

No summary available.

Registry

who.int

Start Date

December 21, 2023

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•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

•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