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Clinical Trial Summary

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients


Clinical Trial Description

We will recruit ß-thalassaemia major patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05864170
Study type Interventional
Source Shenzhen Hemogen
Contact Haigang Sun
Phone 13823168465
Email sunhaigang@genomics.cn
Status Recruiting
Phase Early Phase 1
Start date May 27, 2022
Completion date August 2025

See also
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