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

About

Brief Summary

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

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase III

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
65 Years

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.
  • Screening 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.
  • 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.
  • Orally administered CYP3A4 substrates and MATE and OAT3 substrates are not permitted as concomitant therapy during the administration of losmapimod (defined as baseline visit through end of study treatment).
  • Known active opportunistic or life-threatening infections including 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 patients 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).

Join this Trial

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Study Stats
Protocol No.
22-000970
Category
Neurology
Contact
Jennifer Huynh
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05397470
For detailed technical eligibility, visit ClinicalTrials.gov.