Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
Facioscapulohumeral Muscular Dystrophy (FSHD)
About this trial
This is an interventional treatment trial for Facioscapulohumeral Muscular Dystrophy (FSHD) focused on measuring Facioscapulohumeral muscular dystrophy (FSHD), Facioscapulohumeral muscular dystrophy type 1 (FSHD 1), Facioscapulohumeral muscular dystrophy type 2 (FSHD 2), Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Musculoskeletal Diseases, Neuromuscular Diseases, REACH
Eligibility 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.
- 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).
Sites / Locations
- University of California Irvine
- University of California Los Angeles (UCLA)
- University of Colorado Anschutz Medical Campus
- University of Florida
- University of Kansas Medical Center
- Kennedy Krieger Institute
- University of Massachusetts Memorial Medical Center
- Mayo Clinic
- Washington University School of Medicine
- University of Rochester Medical Center
- Ohio State University Medical Center
- University of Utah
- Virginia Commonwealth University
- University of Washington Medical Center
- University of Calgary
- The Ottawa Hospital Research Institute
- Montreal Neurological Institute and Hospital
- Aarhus Universitetshospital
- Rigshospitalet
- Nice University Hospital - CHU Nice
- Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière
- University Hospital Bonn
- LMU Klinikum Ludwig-Maximilians-Universität München
- Universitätsklinikum Ulm
- Fondazione Serena Onlus- Centro Clinico NEMO
- Fondazione IRCCS Istituto Neurologico Carlo Besta
- Radboudumc
- Leiden University Medical Centre
- Hospital Universitario Donostia
- Hospital Universitario Vall d'Hebron
- Hospital Universitari i Politecnic La Fe
- University College of London Hospitals
- Newcastle upon Tyne NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Placebo Comparator
Experimental
Part A: Placebo-controlled treatment period: Losmapimod
Part A: Placebo-controlled treatment period: Placebo
Part B: Open-label extension
Participants will be randomized to receive losmapimod.
Participants will be randomized to receive placebo
Participants will receive losmapimod, upon completion of all assessments for Part A.