Clinical Trial

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Protocol Amendment History 13 amendments
This ClinicalTrials.gov record has been amended 13 times since 2022-01-27; most recent amendment 2026-02-18.
Status change: Not Yet Recruiting → Recruiting 2023-03-15
Trial Details
NCT Number NCT05230459
Lead Sponsor AskBio Inc
Conditions Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation +1 more
Enrollment 10 participants
Start Date 2023-05-15
Primary Completion 2028-12 (estimated)
Study Completion 2032-12 (estimated)
Updated on ClinicalTrials.gov 2026-02-20