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RECRUITING
NCT03351868
NA

FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector

Sponsor: Shenzhen Geno-Immune Medical Institute

View on ClinicalTrials.gov

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

GENETIC

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