NCT06219239

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

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

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 3 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	KL003 cell injection Drug Product	No conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene

Timeline

• Start date: 2024-01-04
• Primary completion: 2025-12-31
• Completion: 2026-12-31
• First posted: 2024-01-23
• Last updated: 2025-08-05

Locations

1 site across 1 country: China