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RECRUITING
NCT06219239
NA

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

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 transfusion in subjects with transfusion-dependent β-thalassemia.

Official title: Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen

Key Details

Gender

All

Age Range

3 Years - 35 Years

Study Type

INTERVENTIONAL

Enrollment

3

Start Date

2024-01-04

Completion Date

2026-12-31

Last Updated

2025-08-05

Healthy Volunteers

No

Interventions

GENETIC

KL003 cell injection Drug Product

No conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene

Locations (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China