Brief Title
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
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
Brief 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
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 Phase
Phase 1
Study Type
Interventional
Primary Outcome
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
Secondary Outcome
The average Insertion copy number (VCN) in peripheral blood mononuclear cells
Condition
β-Thalassemia Major
Intervention
β-globin restored autologous HSC
Study Arms / Comparison Groups
Experimental
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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
10
Start Date
November 1, 2020
Completion Date
November 30, 2024
Primary Completion Date
November 30, 2022
Eligibility Criteria
Inclusion Criteria:
- 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
post-transplantation.
Exclusion Criteria:
- 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.
Gender
All
Ages
8 Years - 16 Years
Accepts Healthy Volunteers
No
Contacts
Chao Liu, PhD, 13510560664, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT04592458
Organization ID
A-SOP-CT-001
Responsible Party
Sponsor
Study Sponsor
BGI-research
Collaborators
Shenzhen Children's Hospital
Study Sponsor
Chao Liu, PhD, Principal Investigator, BGI-research
Verification Date
September 2020