NCT05442346

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

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

• Status: Suspended
• Phase: N/A
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Bioray Laboratories · Industry
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Thalassemia Major

Interventions

Type	Name	Description
BIOLOGICAL	γ-globin reactivated autologous hematopoietic stem cells	gene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103

Timeline

• Start date: 2023-12-25
• Primary completion: 2024-09-08
• Completion: 2024-11-30
• First posted: 2022-07-05
• Last updated: 2024-02-28

Locations

1 site across 1 country: China