Lentiviral Gene Therapy for CGD
NCT03645486 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10
Last updated 2026-04-24
Summary
This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.
Conditions
- Chronic Granulomatous Disease
Interventions
- GENETIC
-
Infusion of lentiviral TYF-CGD-modified autologous stem cells
Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight
Sponsors & Collaborators
-
Shenzhen Geno-Immune Medical Institute
lead OTHER
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2025-07-01
- Primary Completion
- 2028-06-30
- Completion
- 2029-12-31
Countries
- China
Study Locations
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