NCT05860595

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

• Status: Active Not 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 in subjects with transfusion-dependent β-thalassemia.

Detailed description

Subject participation for this study will be 24 months. Subjects who enroll in this study will be asked to participate in a subsequent 13-year follow-up for gene therapy products.

Conditions

• Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	KL003 cell injection Drug Product	Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene.

Timeline

• Start date: 2023-05-23
• Primary completion: 2025-08-20
• Completion: 2025-10-24
• First posted: 2023-05-16
• Last updated: 2025-08-05

Locations

1 site across 1 country: China