NCT04205435

β-globin Restored Autologous HSC in β-thalassemia Major Patients

a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Bioray Laboratories · Industry
• Sex: All
• Age: 5 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.

Detailed description

β-globin restored autologous hematopoietic stem cells will be manufactured using CRISPR/Cas9 gene editing system. Subject participation for this study will be 1 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.

Conditions

• β-thalassemia Major

Interventions

Type	Name	Description
BIOLOGICAL	β-globin restored autologous HSC	gene edited autologous hematopoietic stem cells with β-globin restoration

Timeline

• Start date: 2021-11-01
• Primary completion: 2022-06-01
• Completion: 2022-07-25
• First posted: 2019-12-19
• Last updated: 2022-10-13

Locations

1 site across 1 country: China