Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

Completed · Phase 2 · Has a placebo group

Conditions studied: Facioscapulohumeral Muscular Dystrophy (FSHD)

In brief

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

Key facts

Study ID: NCT04003974
Run by: Fulcrum Therapeutics
People needed: 80
Starts: 2019-08-09
Expected to finish: 2021-01-28
Last updated by the study team: 2024-07-10

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

The patient must have consented to participate and must have provided signed, dated and witnessed IRB-approved informed consent form that conforms to federal and institutional guidelines.
Male or female subjects
Confirmed diagnosis of FSHD1 with 1 to 9 repeats via assessment of the size of the D4Z4 array on chromosome 4. Genetic confirmation must be obtained prior to the screening MRI and baseline muscle biopsy.
Clinical severity score of 2 to 4 (RICCI Score; Range 0-5), inclusive at screening
Must have a MRI-eligible muscle for biopsy
Must be willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines and other study procedures.
Will practice an approved method of birth control

You may not qualify if…

Has a history of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, a history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; neuromuscular diseases except FSHD (eg, myopathy, neuropathy, neuromuscular junction disorders); or clinically significant history of mental disease.
For subjects who are on drug(s) or supplements that may affect muscle function, as determined by the treating physician, or that are included in the list of drugs presented in the protocol, subjects 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. Changes to the dose or treatment discontinuation during the study can only be done for strict medical reasons by the treating physician with clear documentation and notification to the sponsor.
Acute or chronic history of liver disease or known to have current alanine aminotransferase ≥2 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN, or known history of hepatitis B or C.
Known severe renal impairment (defined as a glomerular filtration rate of <30 mL/min/1.73m2).
Positive screen for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency virus (HIV)-1 and -2.
Male subjects with a female partner who is planning to become pregnant during the study or within 90 days after the last dose of study drug.
Use of another investigational product within 30 days or 5 half-lives (whichever is longer), or according to local regulations, or currently participating in a study with an investigational product. Note: concurrent participation in other non-drug studies may be acceptable if confirmed in writing by the sponsor.

Where it is running

University of California Irvine — Irvine, California, United States
University of California Los Angeles (UCLA) — Los Angeles, California, 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
Washington University School of Medicine — St Louis, Missouri, United States
University of Rochester Medical Center — Rochester, New York, United States
Ohio State University — Columbus, Ohio, United States
University of Utah — Salt Lake City, Utah, United States
Virginia Commonwealth University — Richmond, Virginia, United States
University of Washinton Medical Center — Seattle, Washington, United States
Ottawa Hospital Research Institute — Ottawa, Ontario, Canada
Montreal Neurological Institute and Hospital — Montreal, Quebec, Canada
CHU de NICE- CHU pasteur2 — Nice, France
Hospital de la Sta Creu i St Pau — Barcelona, Spain
Hospital UiP La Fe — Valencia, Spain

Full record on ClinicalTrials.gov

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