Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at Irvine, California and other locations
Dates
study started
estimated completion

Description

Summary

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

Official Title

A Phase 3 Global, 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)

Details

Patients diagnosed with FSHD 1 and FSHD 2 will participate in this study for approximately 53 weeks. This will include a 4-week screening period, a 48-week, placebo-controlled treatment period and a 7 day safety follow-up period. Patients will be randomized to receive 15 mg of losmapimod or placebo twice daily by mouth for 48 weeks.

Keywords

Facioscapulohumeral Muscular Dystrophy (FSHD), FSHD, FSHD 1, FSHD 2, FSH, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Musculoskeletal Diseases, Neuromuscular Diseases, REACH, Fulcrum Therapeutics, Muscular Dystrophy, Facioscapulohumeral, Losmapimod

Eligibility

You can join if…

Open to people ages 18-65

  • 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.

You CAN'T join if...

  • 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.

Locations

  • University of California Irvine accepting new patients
    Irvine California 92868 United States
  • University of California Los Angeles (UCLA) accepting new patients
    Los Angeles California 90095 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Fulcrum Therapeutics
ID
NCT05397470
Phase
Phase 3 Facioscapulohumeral Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 230 study participants
Last Updated