Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
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.
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 16 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date: June 2, 2023
Estimated Primary Completion Date: December 2024
Estimated Study Completion Date: December 2024
Arm:
- Experimental: Cohort 1
- Experimental: Cohort 2
- Experimental: Cohort 3
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 16 |
| Actual Study start date | 02 June 2023 |
| Estimated study completion date | 01 December 2024 |