Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
- Conditions
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- Interventions
- Registration Number
- NCT04264442
- Lead Sponsor
- Fulcrum Therapeutics
- Brief 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.
- Detailed Description
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.
Participation in this open-label extension study will continue until 90 days after 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 76
- 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
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Losmapimod Losmapimod FSHD1 patients with genetic confirmation will receive Losmapimod 15 mg by mouth twice daily for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or until the study is discontinued by the Sponsor.
- Primary Outcome Measures
Name Time Method Number of Participants Reporting Serious Treatment Emergent Adverse Events (Serious TEAEs) and TEAEs Up to 57 months A TEAE is an adverse event that begins on or after the first dose of study drug and on or before the stop of study drug plus 7 days or begins before the first dose of study drug and worsens on or after the first dose of study drug and on or before the stop of study drug plus 7 days. An adverse event with completely missing onset and end dates was considered as TEAE. An adverse event with missing onset date but the end date is on or after the first dose of study drug and before the stop of study drug plus 7 days was considered a TEAE.
Number of Participants With Clinically Significant Changes in Chemistry Parameters Up to 57 months Blood samples were collected for the analysis of chemistry parameters: Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, total bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase and creatine phosphokinase.
Number of Participants With Clinically Significant Changes in Hematology Parameters Up to 57 months Blood samples were collected for the analysis of hematology parameters: hemoglobin (including mean corpuscular volume), mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, hematocrit, red blood cell count, total white blood cell count, platelet count. Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters Up to 57 months Twelve-lead ECGs was performed after participants has been recumbent for at least 5 minutes.
Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters Up to 57 months Blood samples were collected for the analysis of Serum coagulation parameters: International normalized ratio, prothrombin time, partial thromboplastin time.
Number of Participants With Clinically Significant Changes in Urinalysis Up to 57 months Urine samples were collected for the analysis of urinalysis parameters: Leucocytes, blood, nitrite, protein, urobilinogen, bilirubin, pH, specific gravity, ketones, glucose.
Number of Participants With Clinically Significant Changes in Vital Parameters Up to 57 months Vital parameters including pulse rate, respiration rate, blood pressure, and temperature were measured in seated or recumbent for at least 5 minutes. Data for number of participants with abnormal clinically significant changes for vital signs have been presented.
Number of Participants With Clinically Significant Changes in Physical Examinations Up to 57 months Physical examinations included an evaluation of body systems, including but not limited to the following: skin; head, eyes, ears, nose, and throat; respiratory system; cardiovascular system; abdomen (liver, spleen); lymph nodes; neurological system; and musculoskeletal system.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (17)
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
Scroll for more (7 remaining)University of California Irvine🇺🇸Irvine, California, United States