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Clinical Trials in the UK / NCT07678775
Active, not recruiting Phase 3

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

NCT07678775 · tracked via the Priya Life Science UK tracker
Sponsor
ML Bio Solutions, Inc.
Phase
Phase 3
Started
2026-06-05
Last updated
2026-07-02

Condition(s) studied

Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

Investigational drug(s) / intervention(s)

BBP-418 (ribitol)

BBP-418 (ribitol): 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.

Study 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

Sex
ALL
Min age
Max age
Healthy volunteers
No
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 \<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. Exclusion Criteria: * 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.

Primary outcome measure(s)

Trial sites (20)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas
University of California Irvine Medical Center Orange California
University of Colorado Hospital (UCH) - Anschutz Medical Campus Aurora Colorado
University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases Gainesville Florida
University of Iowa Stead Family Children's Hospital Iowa City Iowa
University of Kansas Medical Center (KUMC) Kansas City Kansas
Kennedy Krieger Institute Baltimore Maryland
University of Minnesota Medical School Minneapolis Minnesota
Washington University School of Medicine St Louis Missouri
The Children's Hospital of Philadelphia Philadelphia Pennsylvania
University of Pennsylvania Philadelphia Pennsylvania
Children's Hospital of the Kings Daughters Norfolk Virginia
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
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico Milan Italy
Leids Universitair Medisch Centrum Leiden Netherlands
Universitetssykehuset Nord-Norge, Department of Neurology Tromsø Norway
UCL Great Ormond Street Hospital London United Kingdom
Newcastle University - John Walton Muscular Dystrophy Research Centre Newcastle upon Tyne United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07678775 on ClinicalTrials.gov ↗ ← All trials in the UK