Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
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
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
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.
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
The average Insertion copy number (VCN) in peripheral blood mononuclear cells
β-globin restored autologous HSC
Study Arms / Comparison Groups
Description: 10 transfusion dependent β-thalassemia major subjects who are 8-16 years older will be transplanted with β-globin restored autologous hematopoietic stem cells that are modified with lentiviral vector LentiHBBT87Q encoding the human β-globin gene.
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
November 1, 2020
November 30, 2024
Primary Completion Date
November 30, 2022
- 8-16 years old. Subject and/or subject's legal guardian fully understand and
voluntarily sign informed consent;
- Clinically diagnosed as transfusion-dependent β-thalassemia major;
- With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin
threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3
months before mobilization of hematopoietic stem cell;
- Follow the arrangements for treatment and regular medical checks within two years
- The physical condition does not meet the requirements for hematopoietic stem cell
mobilization and transplantation myeloablation;
- Received gene therapy and allogeneic HSCT in the past.
- Have an available HLA matched donor.
- Enrolling in another clinical trial.
- Other unsuitable conditions identified by doctors.