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Active Not Recruiting
NCT07678775
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
Conditions: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Sex: All
Healthy volunteers: No
Phase: PHASE3
Enrollment: 107
Sponsor: ML Bio Solutions, Inc.
Location: Arkansas Children's Hospital Little Rock Arkansas
Summary
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.
Eligibility Criteria
Inclusion Criteria:
* Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor).
* The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study by giving informed consent (or assent, if \
Source: ClinicalTrials.gov (NCT07678775). StuddyBuddy aggregates publicly available trial information.