Clinical Trial

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

Active, Not Recruiting Phase 1/2
View on ClinicalTrials.gov →
Record status
This record was last updated July 3, 2025 (before its estimated December 2025 completion). Its status may not reflect the trial's current state.
Summary
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 infantile-onset Pompe disease who are younger than 6 months old will be studied.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2023-03-20; most recent amendment 2025-07-01.
Status change: Recruiting → Active, Not Recruiting 2025-07-01
Status change: Not Yet Recruiting → Recruiting 2023-06-04
Trial Details
NCT Number NCT05793307
Lead Sponsor GeneCradle Inc
Conditions Pompe Disease Infantile-Onset
Enrollment 16 participants
Start Date 2023-06-02
Primary Completion 2025-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2025-07-03