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First received: February 16, 2023

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

Study Status: Completed

Recruit Status: RECRUITING

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

Study Type: INTERVENTIONAL


Official Title: 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)

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.

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