BBP-418 (ribitol): Drug: BBP-418. Single arm. Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months.
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.
| Facility | City | Region | Status |
|---|---|---|---|
| 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 | Australia | |
| Rigshospitalet, Neuromuscular Clinic and Research Unit | Copenhagen | Denmark | |
| Charité Universitätsmedizin Berlin and Max Delbrück Center | Berlin | Germany | |
| Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| Leids Universitair Medisch Centrum | Leiden | Netherlands | |
| Universitetssykehuset Nord-Norge, Department of Neurology | Tromsø | Norway | |
| UCL Great Ormond Street Hospital | London | United Kingdom | |
| Newcastle University - John Walton Muscular Dystrophy Research Centre | Newcastle upon Tyne | United Kingdom |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07678775 on ClinicalTrials.gov ↗ ← All trials in the UK