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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Sponsor: Shenzhen Geno-Immune Medical Institute
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
Conditions
Interventions
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