LTE Study of Long Term Effects of BBP-418 in Patients with LGMD2I
Research type
Research Study
Full title
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants with Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
IRAS ID
1013625
Contact name
Alisha Armstrong
Contact email
Sponsor organisation
ML Bio Solutions, Inc.
Research summary
This is an Open-Label Extension Study in patients with limb girdle muscular dystrophy type 2I (LGMD2I) who have previously undergone study 005. The purpose of this study is to determineLong-Term Safety
and Efficacy of BBP-418 (Ribitol) in Participants with LGMD2I/R9.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.
All participants will receive BBP-418 and not the placebo.
Approximately 107 patients (12-60 years old) with LGMD2I will be enrolled into this study.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
26/NE/0040
Date of REC Opinion
23 Mar 2026
REC opinion
Favourable Opinion