Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

• Status: Not yet recruiting
• Trial end date: 2024-03-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a study to evaluate the safety and efficacy of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fulcrum Therapeutics

Criteria

Inclusion Criteria:

• 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.

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.

• 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.