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ACTIVE NOT RECRUITING
NCT05860595
NA

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

Sponsor: Institute of Hematology & Blood Diseases Hospital, China

View on ClinicalTrials.gov

Summary

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells in subjects with transfusion-dependent β-thalassemia.

Key Details

Gender

All

Age Range

3 Years - 35 Years

Study Type

INTERVENTIONAL

Enrollment

3

Start Date

2023-05-23

Completion Date

2025-10-24

Last Updated

2025-08-05

Healthy Volunteers

No

Interventions

GENETIC

KL003 cell injection Drug Product

Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene.

Locations (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China