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

StatusActive, Not Recruiting
PhasePhase 1/2
Started
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.

Record History

  1. Primary completion date: Dec 2024 to Dec 2025 Trial status: Recruiting to Active, Not Recruiting · Completion date: Dec 2024 to Dec 2026 · Site details revised at 5 of 5 sitesv4 Show 3 more changesShow less
    • Trial status: Recruiting to Active, Not Recruiting
    • Completion date: Dec 2024 to Dec 2026
    • Site details revised at 5 of 5 sites
  2. Trial status: Not Yet Recruiting to Recruiting Start date changed from estimate of May 2023 to Jun 2, 2023 · Site details revised at 5 of 5 sitesv3 Show 2 more changesShow less
    • Start date changed from estimate of May 2023 to Jun 2, 2023
    • Site details revised at 5 of 5 sites
  3. Re-verified, no change to tracked fieldsv2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT05793307
Lead Sponsor GeneCradle Inc
Conditions Pompe Disease Infantile-Onset
Enrollment 16 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov