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FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
Sponsor: Shenzhen Geno-Immune Medical Institute
Summary
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
Official title: Gene Transfer for Fanconi Anemia Using a Self-inactivating Lentiviral Vector
Key Details
Gender
All
Age Range
2 Years - 20 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2026-06-01
Completion Date
2030-12-31
Last Updated
2026-06-23
Healthy Volunteers
No
Conditions
Interventions
Gene-modified autologous stem cells
Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances
Locations (1)
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China