Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9 (NCT07678775)
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
🩺 Plain-English Summary (8th-Grade Level)
This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify). Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9. Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.
Who can join: Review the eligibility criteria below.
Location: Arkansas Children's Hospital — Little Rock, Arkansas
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (20 Sites)
- Arkansas Children's Hospital — Little Rock, Arkansas
- University of California Irvine Medical Center — Orange, California
- University of Colorado Hospital (UCH) - Anschutz Medical Campus — Aurora, Colorado
- University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases — Gainesville, Florida
- University of Iowa Stead Family Children's Hospital — Iowa City, Iowa
- University of Kansas Medical Center (KUMC) — Kansas City, Kansas
- Kennedy Krieger Institute — Baltimore, Maryland
- University of Minnesota Medical School — Minneapolis, Minnesota
- Washington University School of Medicine — St Louis, Missouri
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- University of Pennsylvania — Philadelphia, Pennsylvania
- Children's Hospital of the Kings Daughters — Norfolk, Virginia
- Royal Brisbane and Women's Hospital — Brisbane,
- Rigshospitalet, Neuromuscular Clinic and Research Unit — Copenhagen,
- Charité Universitätsmedizin Berlin and Max Delbrück Center — Berlin,
- Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan,
- Leids Universitair Medisch Centrum — Leiden,
- Universitetssykehuset Nord-Norge, Department of Neurology — Tromsø,
- UCL Great Ormond Street Hospital — London,
- Newcastle University - John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.