clinical trial · NCT05230459
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
AskBio Inc·phase2·recruiting·n = 10
Limb Girdle Muscular DystrophyLimb-Girdle Muscular Dystrophy Type 2LGMD2IMuscular DystrophyLGMD2LGMDFKRPFKRP MutationAB-1003 dose level 1AB-1003 dose level 2Placebo
brief 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)
started
May 15, 2023
primary completion
Dec 1, 2028
completion
Dec 1, 2032
last updated
Feb 20, 2026
official title
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)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol