Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
In brief
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.
Key facts
- Study ID
- NCT05775848
- Run by
- ML Bio Solutions, Inc.
- People needed
- 81
- Starts
- 2023-05-31
- Expected to finish
- 2027-07-01
- Last updated by the study team
- 2025-09-29
Who can join
Age: 12 and older, up to 60. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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 — Gainsville, 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 — St 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
Full record on ClinicalTrials.gov
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