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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
107
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.
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California Irvine Medical Center
Orange, California, United States
University of Colorado Hospital (UCH) - Anschutz Medical Campus
Aurora, Colorado, United States
University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, United States
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
...and 10 more locations
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418.
Time frame: 36 months
Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline FVC (forced vital capacity) (percent predicted, performed in a sitting position) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline in PUL 2.0 (performance of the upper limb) total score for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants.
Time frame: 36 months
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