Effects of BBP-418 in Patients with LGMD2I
Research type
Research Study
Full 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)
IRAS ID
1007018
Contact name
Amy Rainey
Contact email
Sponsor organisation
ML Bio Solutions, Inc.
Clinicaltrials.gov Identifier
Research summary
This is a Phase 3, randomized, double-blind, placebo-controlled study in patients with limb girdle muscular dystrophy type 2I (LGMD2I). The purpose of this study is to determine the safety and effectiveness of BBP-418 as a potential treatment of LGMD2I.
LGMD2I is a disease that affects a person’s muscles which gets worse with time and can often make it difficult for some people to walk. In some cases, people will also develop heart and/or breathing problems.
This study is being conducted at approximately 20 locations across the United States, Europe, UK and Australia. Additional regions may also be selected.
This study is a double-blind, placebo-controlled study, which means that neither the patient or the study doctor will know if they are receiving study drug or a placebo.
Participants will be randomly assigned to receive either BBP-418 or placebo and will receive study drug for up to 3 years. After 3 years on the study, they may be given the option to participate in a separate open-label study. In the separate open-label study, all participants will receive BBP-418 and not the placebo.
Approximately 101 patients (12-60 years old) with LGMD2I will be enrolled into this study.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
23/NE/0130
Date of REC Opinion
8 Sep 2023
REC opinion
Further Information Favourable Opinion