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

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Re-verified, no change to tracked fieldsv2
  2. 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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 late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.
Trial Details
NCT Number NCT06391736
Lead Sponsor GeneCradle Inc
Conditions Pompe Disease (Late-onset)
Enrollment 33 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov