A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)

What this Trial Is About

Researchers are evaluating the safety and tolerability of a single intravenous infusion of AB-1003 gene therapy in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This study includes sequential dose-level groups to carefully assess the effects of the treatment. The trial is a two-part, multicenter study with randomized, double-blind, placebo-controlled design to ensure reliable results for adults aged 18 to 65 with confirmed FKRP gene mutations linked to LGMD2I/R9. Participants receive a single intravenous infusion of AB-1003 at one of two dose levels or a placebo in the first part of the study. This dose-escalation phase helps to monitor safety and tolerability before moving to the next phase. The treatment is given as one infusion only, and participants are monitored closely for any side effects or adverse events throughout the study period. During the study, participants will undergo regular evaluations including monitoring for adverse events for up to 52 weeks. Assessments include clinical exams, laboratory tests, and imaging to track the participants’ health and response to the infusion. Safety monitoring is a key focus, with detailed attention to heart function and liver and kidney health. Participants are expected to comply with study procedures and contraception use as required, with the overall study duration spanning approximately one year.

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