Clinical Trials Logo
  1. Home
  2. ß-thalassemia
  3. NCT05745532

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

ß-thalassemia Clinical Trial

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

Clinical Trial 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

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05745532
Study type Interventional
Source Shenzhen Hemogen
Contact Haigang Sun
Phone 13823168465
Email sunhaigang@genomics.cn
Status Recruiting
Phase Early Phase 1
Start date December 1, 2020
Completion date May 30, 2025
View Details
See also
  Status Clinical Trial Phase
Completed NCT00560820 - Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload Phase 1
Recruiting NCT05864170 - the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia Early Phase 1
Completed NCT04054921 - Safety Study for Beta Thalassemia Subjects on PTG-300 Phase 2
Not yet recruiting NCT05773729 - Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia N/A
Not yet recruiting NCT05776173 - Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia N/A
Completed NCT03802201 - Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia Phase 2
Not yet recruiting NCT03358498 - Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox N/A
Recruiting NCT05494333 - Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia