Clinical Trial

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

Recruiting
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Record status
This record was last updated June 14, 2024 (before its estimated March 2026 completion). Its status may not reflect the trial's current state.
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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2023-03-15.
Status change: Not Yet Recruiting → Recruiting 2024-06-13
Trial Details
NCT Number NCT05773729
Lead Sponsor Shanghai BDgene Co., Ltd.
Collaborators: Shanghai Children's Medical Center
Conditions β-thalassemia
Enrollment 10 participants
Start Date 2023-09-15
Primary Completion 2026-03 (estimated)
Study Completion 2026-10 (estimated)
Updated on ClinicalTrials.gov 2024-06-14