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drugset / Trial / NCT05773729

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia

NCT05773729 ↗

Recruiting 10 enrolled Shanghai BDgene Co., Ltd. Shanghai Children's Medical Center · collab
NaSingle-groupOpen-labelTreatment

Summary

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Timeline

Start
2023-09-15
Primary completion
2026-03
Completion
2026-10

Drugs

EvaluationDrugModalityDoseRoute
Subject BD211 Cell therapy 5e+06 cells/kg Intravenous
Subject BD211 Cell therapy 1e+07 cells/kg Intravenous

Indications

beta thalassemia