Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Condition(s) studied
Investigational drug(s) / intervention(s)
GC301: GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene
Study 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 late-onset Pompe disease (LOPD) who are ≥ 6 years old will be studied.
Eligibility
Primary outcome measure(s)
- Number of Participants With Adverse Events — 52 weeks
Number of Participants with Adverse Events as a Measure of Safety and Tolerability - Dose-limiting toxicity (DLT) rate based on protocol-specific adverse events (Phase 1) — within 30 days after treatment
- Percent Predicted Upright Forced Vital Capacity (FVC)(Phase 2) — 52 weeks
Change from baseline in percentage of predicted FVC measured by pulmonary function testing
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Chinese PLA General Hospital | Beijing | China | Recruiting |
More GeneCradle Inc trials in China
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06391736 on ClinicalTrials.gov ↗ ← All trials in China