NCT04264442

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 76 (actual)
• Sponsor: Fulcrum Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is an open-label extension to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.

Detailed description

This study is an open-label extension study to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study. This study is a multi-center clinical trial. It will be conducted in North America, Canada and Europe. Only patients who participated and competed all study procedures in the ReDUX4 Study treatment period will be eligible to participate in this open label extension study. Patients who complete the randomized, placebo-controlled portion of the study will have the option to roll over into the open-label extension study. Patients will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. All patients will attend clinic visits approximately every 12 weeks. Participation in this open-label extension study will continue until 90 days after losmapimod becomes commercially available, the patient withdraws from the study, or the Sponsor decides to close the study. The primary endpoint of the study is to evaluate the safety and tolerability of long-term dosing of losmapimod in patients with FSHD.

Conditions

• Facioscapulohumeral Muscular Dystrophy (FSHD)

Interventions

Type	Name	Description
DRUG	Losmapimod	Patients will receive Losmapimod 15 mg by mouth twice daily for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or until the study is discontinued by the Sponsor. The study drug should be taken with food and the date and time of each dose taken should be recorded in the subject diary.

Timeline

• Start date: 2020-02-13
• Primary completion: 2024-11-07
• Completion: 2024-11-07
• First posted: 2020-02-11
• Last updated: 2025-09-30
• Results posted: 2025-09-30

Locations

17 sites across 4 countries: United States, Canada, France, Spain

Regulatory

• FDA-regulated drug study