Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9

Running, not enrolling · Phase 3

Conditions studied: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

In brief

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.

Key facts

Study ID

NCT07678775

Run by

ML Bio Solutions, Inc.

People needed

107

Starts

2026-06-05

Expected to finish

2030-10-01

Last updated by the study team

2026-07-02

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 <18 years of age).
• Is willing and able to complete all study procedures according to the Schedule of Assessments.
• A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception from the time of consent through 30 days after the last dose of study drug in this study.

You may not qualify if…

• Has developed clinically significant concomitant disease that would, in the Investigator's opinion, be likely to unfavorably impact study participation, including:
• Any significant concomitant medical condition, including psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9
• Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding
• Is pregnant (based on the Baseline / Day 1 pregnancy test result) and/or breastfeeding or planning to conceive children within the projected duration of the study through 30 days after the last dose of study drug in this study.
• Has active suicidal ideation, defined as having a suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS at Baseline / Day 1.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• University of California Irvine Medical Center — Orange, California, United States
• Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan, Italy
• Leids Universitair Medisch Centrum — Leiden, Netherlands
• 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
• Universitetssykehuset Nord-Norge, Department of Neurology — Tromsø, Norway
• 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, Australia
• Rigshospitalet, Neuromuscular Clinic and Research Unit — Copenhagen, Denmark
• Charité Universitätsmedizin Berlin and Max Delbrück Center — Berlin, Germany
• UCL Great Ormond Street Hospital — London, United Kingdom
• Newcastle University - John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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