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
ClinicalTrials.gov ID: NCT05793307
Sponsor: GeneCradle Inc
Information provided by: GeneCradle Inc (Responsible Party)
Last Update Posted: 2023-06-06
Brief 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.
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
Intervention / Treatment:
- Genetic: GC301
| Category | Value |
|---|---|
| Study Start (Actual) |
2023-06-02
|
| Primary Completion (Estimated) |
2024-12-01
|
| Study Completion (Estimated) |
2024-12-01
|
| Enrollment (Estimated) | 16 |
| Study Type | Interventional |
| Phase | Phase 1 Phase 2 |
| Other Study ID Numbers |
JLJY-GC301-IOPD-001
|