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

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