Summary

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

Description

Summary

This study is an open-label extension to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.

Official Title

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

Details

This study is an open-label extension study to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.

This study is a multi-center clinical trial. It will be conducted in North America, Canada and Europe. Only patients who participated and competed all study procedures in the ReDUX4 Study treatment period will be eligible to participate in this open label extension study.

Patients who complete the randomized, placebo-controlled portion of the study will have the option to roll over into the open-label extension study.

Patients will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. All patients will attend clinic visits approximately every 12 weeks and 7 days after the last dose of study drug for safety follow-up assessment.

Participation in this open-label extension study will continue until losmapimod becomes commercially available, the patient withdraws from the study, or the Sponsor decides to close the study.

The primary endpoint of the study is to evaluate the safety and tolerability of long-term dosing of losmapimod in patients with FSHD.

Keywords

Facioscapulohumeral Muscular Dystrophy (FSHD) FSHD, FSHD1 Muscular Dystrophy Muscular Dystrophies Facioscapulohumeral Muscular Disorders Musculoskeletal Diseases Neuromuscular Diseases Muscular Dystrophy, Facioscapulohumeral Losmapimod

Eligibility

You can join if…

Open to people ages 18-65

  • The patient must have consented to participate and must have provided signed, dated and witnessed an IRB-approved informed consent form that conforms to federal and institutional guidelines.
  • Male or female subjects
  • Patients must be between 18 and 65 years of age, inclusive
  • 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 will 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 CAN'T join 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.

Locations

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

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Fulcrum Therapeutics
ID
NCT04264442
Phase
Phase 2
Study Type
Interventional
Last Updated