Ribitol for Limb-Girdle Muscular Dystrophy
(Fortify Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial will test the safety and effectiveness of BBP-418 in patients aged 12 to 60 who have a specific type of muscular dystrophy called LGMD2I/R9. The goal is to see if this new treatment can help improve their condition over several months.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot use ribose or other sugar alcohol supplements, systemic corticosteroids for muscular dystrophy, or be on experimental therapies within 90 days before the screening. It's best to discuss your current medications with the study team.
Is ribitol safe for use in humans?
What makes the drug Ribitol unique for treating Limb-Girdle Muscular Dystrophy?
Ribitol is unique because it works by increasing the levels of a specific sugar molecule called matriglycan, which is important for muscle function, and it compensates for the decreased function of a mutated protein in the disease. Unlike other treatments, it is a simple sugar alcohol that can be administered orally and has shown to improve muscle function and lifespan in animal models with minimal side effects.12346
What data supports the effectiveness of the drug BBP-418 (Ribitol) for treating Limb-Girdle Muscular Dystrophy?
Research shows that ribitol can improve muscle function and increase lifespan in mouse models of Limb-Girdle Muscular Dystrophy by enhancing the expression of a protein called matriglycan, which is important for muscle health. Additionally, combining ribitol with gene therapy has shown promising results in improving muscle pathology in similar conditions.12345
Are You a Good Fit for This Trial?
This trial is for individuals aged 12-60 with genetically confirmed LGMD2I/R9, weighing over 30 kg. They must understand and consent to study procedures, use effective contraception if of childbearing or reproductive potential, and be able to complete all study tasks like biopsies. Exclusions include significant other diseases, severe kidney issues, recent surgeries affecting assessments, pregnancy/breastfeeding intentions within the study period through 12 weeks after last dose.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive BBP-418 or placebo for long-term administration
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- BBP-418 (Ribitol)
Find a Clinic Near You
Who Is Running the Clinical Trial?
ML Bio Solutions, Inc.
Lead Sponsor