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

ß-Thalassemia Major Clinical Trial

Official title:
A Single Center, Open Label Study to Evaluate the Safety and Efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

Status Not yet recruiting

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

Subjects with ß-Thalassemia major will be recruited and their autologous hematopoietic stem cells will be collected and modified with LentiHBBT87Q system to restore the β-globin expression. After conditioning, the β-globin restored autologous hematopoietic stem cells will be infused back to patients, and a 2 years follow up visit will be conducted and the data will be collected. Participants in this study will be also asked to participant in a subsequent follow up study that will monitor the long-term safety and efficacy of the treatment for up to 13 years post-transplantation. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04592458
Study type	Interventional
Source	BGI-research
Contact	Jing Li, PhD
Phone	13510560664
Email	lijing4@genomics.cn
Status	Not yet recruiting
Phase	Phase 1
Start date	November 1, 2020
Completion date	November 30, 2024

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT00390858` - A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.	Phase 2
Active, not recruiting	`NCT01846923` - B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic Individuals	Phase 4
Terminated	`NCT04205435` - β-globin Restored Autologous HSC in β-thalassemia Major Patients	Phase 1/Phase 2
Withdrawn	`NCT01724138` - An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old	Phase 4
Completed	`NCT01745120` - A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants	Phase 1/Phase 2
Recruiting	`NCT05776173` - Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia	N/A