Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
NCT ID: NCT05397470
Last Updated: 2025-11-10
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
260 participants
INTERVENTIONAL
2022-06-16
2024-11-19
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part A: Placebo-controlled treatment period: Losmapimod
Participants will be randomized to receive losmapimod.
Losmapimod
Losmapimod 15 mg will be administered BID by mouth along with food.
Part A: Placebo-controlled treatment period: Placebo
Participants will be randomized to receive placebo
Placebo oral tablet
Placebo will be administered BID by mouth along with food.
Part B: Open-label extension
Participants will receive losmapimod, upon completion of all assessments for Part A.
Losmapimod
Losmapimod 15 mg will be administered BID by mouth along with food.
Interventions
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Losmapimod
Losmapimod 15 mg will be administered BID by mouth along with food.
Placebo oral tablet
Placebo will be administered BID by mouth along with food.
Eligibility Criteria
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Inclusion Criteria
* Genetically confirmed diagnosis of FSHD 1 or FSHD 2.
* Clinical severity score of 2 to 4 (Ricci Score; Range 0-5), at screening. Participants who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study.
* Screening total RSA (Q1-Q4) without weight in the dominant UE assessed by RWS ≥ 0.2 and ≤ 0.7.
* No contraindications to MRI.
Exclusion Criteria
* Participants who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator: participants 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 Human Immunodeficiency virus (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 participants 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).
18 Years
65 Years
ALL
No
Sponsors
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Fulcrum Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of California Irvine
Irvine, California, United States
University of California Los Angeles (UCLA)
Los Angeles, California, United States
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
University of Florida
Gainesville, Florida, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
University of Rochester Medical Center
Rochester, New York, United States
Ohio State University Medical Center
Columbus, Ohio, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
University of Calgary
Calgary, Alberta, Canada
The Ottawa Hospital Research Institute
Ottawa, Ontario, Canada
Montreal Neurological Institute and Hospital
Montreal, Quebec, Canada
Aarhus Universitetshospital
Aarhus, , Denmark
Rigshospitalet
Copenhagen, , Denmark
Nice University Hospital - CHU Nice
Nice, PACA, France
Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière
Paris, , France
University Hospital Bonn
Bonn, , Germany
LMU Klinikum Ludwig-Maximilians-Universität München
München, , Germany
Universitätsklinikum Ulm
Ulm, , Germany
Fondazione Serena Onlus- Centro Clinico NEMO
Milan, Lombardy, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Radboudumc
Nijmegen, Gelderland, Netherlands
Leiden University Medical Centre
Leiden, Southern Holland, Netherlands
Hospital Universitario Donostia
San Sebastián, Guipuzkoa, Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
University College of London Hospitals
London, , United Kingdom
Newcastle upon Tyne NHS Foundation Trust
Newcastle upon Tyne, , United Kingdom
Countries
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References
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Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.
Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.
Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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1821-FSH-301
Identifier Type: -
Identifier Source: org_study_id