NCT04211480

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: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Bioray Laboratories · Industry
• Sex: All
• Age: 5 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to 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 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	γ-globin reactivated autologous hematopoietic stem cells	gene edited autologous hematopoietic stem cells with γ-globin expression

Timeline

• Start date: 2020-10-01
• Primary completion: 2023-10-15
• Completion: 2023-11-27
• First posted: 2019-12-26
• Last updated: 2024-01-05

Locations

1 site across 1 country: China