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Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

Thalassemia Major Clinical Trial

Official title:
an Open Label Trial of Evaluation of the Safety and Efficacy of Treatment With γ-globin Reactivated Autologous Hematopoietic Stem Cells in Subjects With β-thalassemia Major

Clinical Trial Summary

This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Clinical Trial Description

γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05442346
Study type Interventional
Source Bioray Laboratories
Contact
Status Suspended
Phase N/A
Start date December 25, 2023
Completion date November 30, 2024
View Details
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