drugset / Trial / NCT05567627
Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
Unknown
6 enrolled
Seventh Medical Center of PLA General Hospital
GeneCradle Therapeutics, Inc · collab
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2022-08-01
- Primary completion
- 2024-09
- Completion
- 2025-09
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GC301 | Gene therapy (AAV / viral vector) | 1.2e+14 vg/kg | Intravenous |