Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
- Conditions
- Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
- Interventions
- Drug: BBP-418 (ribitol)Other: Placebo
- Registration Number
- NCT05775848
- Lead Sponsor
- ML Bio Solutions, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 81
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description BBP-418 BBP-418 (ribitol) BBP-418 Granules for Oral Solution will be supplied as granules in tri-ply PET/Aluminum/PE sachets for unit dose. The number of sachets to reconstitute will depend on the applicable dose to be delivered, 9 g BID or 12 g BID, as determined by the weight of the participant. The granules will be reconstituted in water for oral administration. BBP-418 Placebo BBP-418 Granules for Oral Solution will be supplied as granules in tri-ply PET/Aluminum/PE sachets for unit dose. The number of sachets to reconstitute will depend on the applicable dose to be delivered, 9 g BID or 12 g BID, as determined by the weight of the participant. The granules will be reconstituted in water for oral administration. Placebo to Match BBP-418 BBP-418 (ribitol) The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions. Placebo to Match BBP-418 Placebo The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions.
- Primary Outcome Measures
Name Time Method Frequency and severity of treatment-emergent adverse events following 36 months of treatment to assess safety of BBP-418 or placebo 36 months Change from baseline in North Star Assessment for Limb Girdle Muscular Dystrophy following 36 months of treatment to assess efficacy of BBP-418 or placebo 36 months
- Secondary Outcome Measures
Name Time Method Change from baseline in 10 meter walk test velocity to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9 36 months Change from baseline in pulmonary function as measured by FVC (percent predicted, performed in a sitting position) to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9 36 months Change from baseline in the Performance of Upper Limb scale 2.0 to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9 36 months
Trial Locations
- Locations (21)
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University of California Irvine
🇺🇸Irvine, California, United States
University of Colorado Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
University of Minnesota, Twin Cities
🇺🇸Minneapolis, Minnesota, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
🇺🇸Portland, Oregon, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of the Kings Daughters
🇺🇸Norfolk, Virginia, United States
Royal Brisbane and Women's Hospital,
🇦🇺Brisbane, Queensland, Australia
Rigshospitalet, Neuromuscular Clinic and Research Unit
🇩🇰Copenhagen, Denmark
Charité Universitätsmedizin Berlin and Max Delbrück Center
🇩🇪Berlin, Germany
IRCCS Ca' Granda Ospedale
🇮🇹Milan, Italy
Leiden University Medical Center
🇳🇱Leiden, Netherlands
Universitetssykehuset Nord-Norge, Department of Neurology
🇳🇴Tromsø, Norway
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
International Centre for Life
🇬🇧Newcastle Upon Tyne, United Kingdom