efficacy and safety of losmapimod in treating participants with facioscapulohumeral muscular dystrophy (fshd) (reach)

Conditions: Facioscapulohumeral Muscular Dystrophy (FSHD)

Summary: this is a study to evaluate the safety and efficacy of losmapimod in treating participants with facioscapulohumeral muscular dystrophy (fshd). participants diagnosed with facioscapulohumeral muscular dystrophy type 1 (fshd1) or facioscapulohumeral muscular dystrophy type 2 (fshd2) will participate in part a (placebo-controlled treatment period) and will be randomized in a 1:1 ratio to receive losmapimod 15 milligrams (mg) or placebo orally twice daily (bid). upon completion of part a, participants will have the option to rollover into part b (open-label extension) to evaluate the long-term safety, tolerability, and efficacy of losmapimod and will receive losmapimod 15 mg orally bid.


Eligibility criteria:
inclusion criteria: * participants must be between 18 and 65 years of age, inclusive. * genetically confirmed diagnosis of fshd 1 or fshd 2. * clinical severity score of 2 to 4 (ricci score; range 0-5), at screening. participants who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study. * screening total rsa (q1-q4) without weight in the dominant ue assessed by rws ≥ 0.2 and ≤ 0.7. * no contraindications to mri. exclusion criteria: * previously diagnosed cancer that has not been in complete remission for at least 5 years. localized carcinomas of the skin and carcinoma in situ of the cervix that have been resected or ablated for cure are not exclusionary. * participants who are on drug(s) or supplements that may affect muscle function, as determined by the investigator: participants must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study. * known active opportunistic or life-threatening infections including human immunodeficiency virus (hiv) and hepatitis b or c. * known active or inactive tuberculosis infection. * acute or chronic history of liver disease. * known severe renal impairment. * history of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ecgs. * use of another investigational product within 30 days or 5 half-lives (whichever is longer) or currently participating in a study of an investigational device. * current or anticipated participation in a natural history study. previous participation is allowed but participants cannot continue after enrollment in study 1821-fsh-301. * known hypersensitivity to losmapimod or any of its excipients. * previous participation in a fulcrum-sponsored fshd losmapimod study (fis-001-2019 or fis-002-2019). note that all other inclusion and exclusion criteria are listed in the protocol and only key are presented.

Keywords: facioscapulohumeral muscular dystrophy (fshd) facioscapulohumeral muscular dystrophy type 1 (fshd 1) facioscapulohumeral muscular dystrophy type 2 (fshd 2) muscular dystrophies muscular dystrophy facioscapulohumeral muscular disorders musculoskeletal diseases neuromuscular diseases reach