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NOT YET RECRUITING
NCT03351829
NA

Gene Therapy of Beta Thalassemia Using a 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 transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Key Details

Gender

All

Age Range

4 Years - 70 Years

Study Type

INTERVENTIONAL

Enrollment

20

Start Date

2026-12-31

Completion Date

2030-12-31

Last Updated

2026-08-27

Healthy Volunteers

No

Interventions

GENETIC

Gene-modified autologous hematopoeitic stem cells

1 infusion of 5x10\^6\~1x10\^7 per kilogram body weight gene-modified cells; or more infusions depending on the circumstances

Locations (1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China