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Study identifier: NCT05773729 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: β-thalassemia  ·  Sponsor: Shanghai BDgene Co., Ltd.

PhaseNA
Planned participants10
Who can joinAll sexes, 3 Years to 18 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Chen Jing, M.D.  ·  +86-18930830632  ·  chenjing@scmc.com.cn

Always discuss trial participation with your own doctor first.

Locations (1)

Shanghai Children's Medical CentreShanghai, ChinaRecruiting

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Source record: clinicaltrials.gov/study/NCT05773729