NCT05230459

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

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)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: AskBio Inc · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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)

Conditions

• Limb Girdle Muscular Dystrophy
• Limb-Girdle Muscular Dystrophy Type 2
• LGMD2I
• Muscular Dystrophy
• LGMD2
• LGMD
• FKRP
• FKRP Mutation
• Fukutin Related Protein

Interventions

Type	Name	Description
GENETIC	AB-1003 dose level 1	Single intravenous infusion of AB-1003 gene therapy at dose level 1
GENETIC	AB-1003 dose level 2	Single intravenous infusion of AB-1003 gene therapy at dose level 2
OTHER	Placebo	Single intravenous infusion of Placebo

Timeline

• Start date: 2023-05-15
• Primary completion: 2028-12-01
• Completion: 2032-12-01
• First posted: 2022-02-09
• Last updated: 2026-02-20

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study