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Clinical Trial Summary

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.


Clinical Trial Description

CS-101 is an autologous CD34+ cell suspension modified by ex vivo base editing technology, removing the inhibitory effect of BCL11A on the γ-globin coding gene, inducing the production of γ-globin chains, increasing the concentration of fetal hemoglobin (HbF) in the blood, compensating for the loss of adult hemoglobin (HbA) to treat transfusion-dependent type/ Major β - thalassemia. The therapy addresses two major challenges in the treatment of the disease: lack of matching donors and graft-versus-host responses commonly seen in allogeneic hematopoietic stem cell transplantation. The study consists of the following five phases: Screening phase: Sign informed consent, complete screening assessments, and confirm the eligibility for enrollment; Baseline: check the subject's baseline status; Mobilization, collection and manufacturing phase: mobilize, collect autologous CD34+ cells and manufacture, release and transport CS-101 product; Conditioning and treatment phase: including myeloablation and CS-101 product infusion; Follow-up phase: 180 days post-infusion. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06065189
Study type Interventional
Source Children's Hospital of Fudan University
Contact Xiaowen Zhai, M.D.
Phone +86-021-64931126
Email zhaixiaowendy@163.com
Status Recruiting
Phase Early Phase 1
Start date November 7, 2023
Completion date December 31, 2024

See also
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