Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) (NCT05775848)
A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
🩺 Plain-English Summary (8th-Grade Level)
This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established epidemiology of the disease.
Who can join: Review the eligibility criteria below.
Location: Arkansas Children's Hospital — Little Rock, Arkansas
Age range: 12 Years to 60 Years
🧪 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 (21 Sites)
- Arkansas Children's Hospital — Little Rock, Arkansas
- University of California Irvine — Irvine, California
- University of Colorado Anschutz Medical Campus — Aurora, Colorado
- University of Florida — Gainsville, Florida
- University of Iowa — Iowa City, Iowa
- University of Kansas Medical Center — Kansas City, Kansas
- Kennedy Krieger Institute — Baltimore, Maryland
- University of Minnesota, Twin Cities — Minneapolis, Minnesota
- Washington University School of Medicine — St Louis, Missouri
- Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront — Portland, Oregon
- 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, Queensland
- Rigshospitalet, Neuromuscular Clinic and Research Unit — Copenhagen,
- Charité Universitätsmedizin Berlin and Max Delbrück Center — Berlin,
- IRCCS Ca' Granda Ospedale — Milan,
- Leiden University Medical Center — Leiden,
- Universitetssykehuset Nord-Norge, Department of Neurology — Tromsø,
- Great Ormond Street Hospital for Children — London,
- International Centre for Life — 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.
More studies in Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
- Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9 — Little Rock, Arkansas
Explore by condition: All Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) clinical trials
💡 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.