Home » Clinical Trials » Muscular dystrophy » NCT05230459
NCT05230459

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)

Study 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)

Want to learn more about this trial?

Request More Info

Interventions

AB-1003 dose level 1GENETIC
Single intravenous infusion of AB-1003 gene therapy at dose level 1
AB-1003 dose level 2GENETIC
Single intravenous infusion of AB-1003 gene therapy at dose level 2
PlaceboOTHER
Single intravenous infusion of Placebo

Study Locations

FacilityCityStateCountry
University of California - IrvineIrvineCaliforniaUnited States
University of IowaIowa CityIowaUnited States
University of Kansas Medical CenterKansas CityKansasUnited States
Kennedy Krieger InstituteBaltimoreMarylandUnited States
VCURichmondVirginiaUnited States
University of Washington Medical CenterSeattleWashingtonUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026