NCT05793307

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

A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: GeneCradle Inc · Industry
• 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

• Pompe Disease Infantile-Onset

Interventions

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

Timeline

• Start date: 2023-06-02
• Primary completion: 2025-12-01
• Completion: 2026-12-01
• First posted: 2023-03-31
• Last updated: 2025-07-03

Locations

5 sites across 1 country: China