Losmapimod for Facioscapulohumeral Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and tolerance of long-term use of Losmapimod, a treatment for Facioscapulohumeral Muscular Dystrophy (FSHD1). FSHD1 is a genetic muscle disorder that weakens the face, shoulders, and upper arms. Participants will take Losmapimod pills twice daily. This trial suits those with FSHD1 confirmed by genetic testing and who participated in the ReDux4 study. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
You may need to stay on a stable dose of any medications or supplements that affect muscle function for at least 3 months before starting the study and continue on that dose during the study. Any changes to your medication can only be made for strict medical reasons.
Is there any evidence suggesting that Losmapimod is likely to be safe for humans?
Research has shown that Losmapimod is generally well-tolerated by over 3,600 people in various studies. Although these studies did not specifically target rare or muscular conditions, this broad use suggests it may be safe for many individuals. In studies focused on facioscapulohumeral muscular dystrophy (FSHD), researchers also assessed Losmapimod for safety. The results indicated it was well-tolerated, with most participants not experiencing severe side effects.
This trial examines long-term use, so researchers are closely monitoring safety over an extended period. The progression of Losmapimod to this stage of testing suggests that earlier studies did not identify major safety issues. However, like any treatment, some side effects may occur, so discussing any concerns with the study team is important.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Facioscapulohumeral Muscular Dystrophy (FSHD), which mainly focus on managing symptoms, Losmapimod works by targeting a specific protein pathway linked to muscle inflammation and degeneration. This drug is taken orally at a dose of 15 mg twice daily, offering a convenient administration method compared to other treatments that might require injections or infusions. Researchers are excited about Losmapimod because it represents a novel approach that could potentially slow down or alter the disease progression, offering hope for more effective management of FSHD.
What evidence suggests that Losmapimod might be an effective treatment for FSHD1?
Research has shown that Losmapimod, the treatment under study in this trial, may help treat facioscapulohumeral muscular dystrophy (FSHD). In earlier studies, patients experienced improvements in muscle strength and abilities, such as reach distance. Specifically, one study found a 9.63% increase in shoulder strength, which is crucial for muscle function. Losmapimod has also been associated with better muscle size and quality. While these results are promising, further research is needed to understand its long-term benefits for FSHD.14678
Who Is on the Research Team?
Marie-Helene Jouvin, MD
Principal Investigator
Fulcrum Therapeutics
Are You a Good Fit for This Trial?
This trial is for adults aged 18-65 with Facioscapulohumeral Muscular Dystrophy (FSHD) who were in the ReDux4 study. Participants must be able to follow the study plan and use approved birth control methods. Those with illnesses that could affect results or pose risks, allergies, heart or brain diseases, other muscle disorders, or significant mental illness cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg daily
Open-label extension
Participants continue to receive losmapimod until 90 days after it becomes commercially available or the study is closed
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Losmapimod
Find a Clinic Near You
Who Is Running the Clinical Trial?
Fulcrum Therapeutics
Lead Sponsor