NCT05775848

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 81 (estimated)
• Sponsor: ML Bio Solutions, Inc. · Industry
• Sex: All
• Age: 12 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

Interventions

Type	Name	Description
DRUG	BBP-418 (ribitol)	The BBP-418 drug product is provided as Granules for Oral solution consisting of BBP-418 drug substance and silicon dioxide in a multilaminate sachet with a foil barrier. Silicon dioxide is generally regarded as safe and listed in the FDA IIAD. Silicon dioxide is a compendial excipient and is commonly used in pharmaceutical dosage forms. The BBP-418 Granules for Oral Solution are provided in sachets.The BBP-418 Granules for Oral Solution are reconstituted in water for oral administration.
OTHER	Placebo	A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product.

Timeline

• Start date: 2023-05-31
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2023-03-20
• Last updated: 2025-09-29

Locations

21 sites across 8 countries: United States, Australia, Denmark, Germany, Italy, Netherlands, Norway, United Kingdom

Regulatory

• FDA-regulated drug study