Open Actively Recruiting
Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
This is a study to evaluate the safety and efficacy of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.
- Patients 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. Patients who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study.
- Baseline total relative surface area (RSA) (Q1-Q4) without weight in the dominant upper extremities (UE) assessed by reachable workspace (RWS) ≥ 0.2 and ≤ 0.7.
- At least one short tau inversion recovery (STIR) positive muscle in the lower extremities.
- No contraindications to MRI.
- 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.
- Patients who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator: patients 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 HIV and hepatitis B or C.
- 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.
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- UCLA Westwood