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

Recruiting N/A Interventional Study
β-thalassemia
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
3 – 18
Sex
Any
Study type
Interventional
Purpose
Treatment
Participants needed
10 (estimated)
Sponsor
Shanghai BDgene Co., Ltd. · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Ages 3 to 18 years old, including: The parents or legal guardians must be able to understand and provide ICFs. If available, it is strongly recommended that children aged ≥8 years in treatment decisions and obtain written ICFs and be clearly documented; Diagnosed as Trans…
Contacts

Chen Jing, M.D.

+86-18930830632

chenjing@scmc.com.cn

CONTACT