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Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
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.
Details
| Lead sponsor | Institute of Hematology & Blood Diseases Hospital, China |
|---|---|
| Phase | NA |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 3 |
| Start date | Tue May 23 2023 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Fri Oct 24 2025 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- Transfusion-dependent Beta-Thalassemia
Interventions
- KL003 cell injection Drug Product
Countries
China