NCT05745532

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Shenzhen Hemogen · Industry
• Sex: All
• Age: 8 Years – 16 Years
• Healthy volunteers: Not accepted

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

Detailed 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.

Conditions

• β-thalassemia

Interventions

Type	Name	Description
BIOLOGICAL	β-globin restored autologous hematopoietic stem cells	β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Timeline

• Start date: 2020-12-01
• Primary completion: 2025-05-30
• Completion: 2025-12-30
• First posted: 2023-02-27
• Last updated: 2024-11-29

Locations

1 site across 1 country: China