NCT05567627

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Seventh Medical Center of PLA General Hospital · Academic / Other
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Infantile-onset Pompe Disease

Interventions

Type	Name	Description
BIOLOGICAL	Genetic: GC301	GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Timeline

• Start date: 2022-08-01
• Primary completion: 2024-09-01
• Completion: 2025-09-01
• First posted: 2022-10-05
• Last updated: 2023-11-01

Locations

1 site across 1 country: China