Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.

NCT Number	NCT06391736
Lead Sponsor	GeneCradle Inc
Conditions	Pompe Disease (Late-onset)
Enrollment	33 participants
Start Date	2024-04-19
Primary Completion	2026-12 (estimated)
Study Completion	2026-12 (estimated)
Updated on ClinicalTrials.gov	2025-07-03