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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05745532
Recruitment Status : Recruiting
First Posted : February 27, 2023
Last Update Posted : February 27, 2023
Sponsor:
Information provided by (Responsible Party):
Shenzhen Hemogen

Study Description
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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

Condition or disease Intervention/treatment Phase
β-thalassemia Biological: β-globin restored autologous hematopoietic stem cells Early Phase 1

Detailed 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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Actual Study Start Date : December 1, 2020
Estimated Primary Completion Date : May 30, 2023
Estimated Study Completion Date : May 30, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Experimental
Ten transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q.
 Biological: β-globin restored autologous hematopoietic stem cells
β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q


Outcome Measures
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Primary Outcome Measures :
  1. Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: 0-100 days ]
    The number and the percentage of adverse events related to transplantation in 100 days will be summarized according to NCI CTCAE 5.0

  2. Overall survival [ Time Frame: 0-24 months ]
    Number of patients alive through the whole trial will be record

  3. Proportion of engraftment [ Time Frame: 0-24 months ]
    Neutrophil count [ANC] >=500 /mm3 for 3 consecutive days and platelet count [PLT] >20,000/mm3 for7 consecutive days

  4. Replication competent lentivirus (RCL) [ Time Frame: 0-24 months ]
    The percentage of RCL should be negative in the 24 months after transplant

  5. Dynamics of viral integration sites (VIS) [ Time Frame: 0-24 months ]
    Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment . More than 1000 VIS retrieved from peripheral blood should be checked.


Secondary Outcome Measures :
  1. The average Insertion copy number (VCN) in peripheral blood mononuclear cells [ Time Frame: 18-24 Months ]
    The average insertion copy number (VCN) should be ≥0.1 in peripheral blood mononuclear cells

  2. The expression level of exogenous adult hemoglobin [ Time Frame: 18-24 Months ]
    Exogenous adult hemoglobin will be evaluated by globin chains and hemoglobin synthesis on peripheral blood by HPLC and the exogenous adult hemoglobin level is ≥2.0g/dL

  3. Change from baseline in annualized frequency of packed RBC transfusions [ Time Frame: 18-24 Months ]
    Compare the annualized number of pRBC transfusions before gene therapy with the Month 18 and Month 24 period after transplant, the percentage change will be recorded


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05745532


Contacts
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Contact: Haigang Sun 13823168465 sunhaigang@genomics.cn

Locations
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China, Guangdong
Shenzhen Children's Hospital Recruiting
Shenzhen, Guangdong, China, 518083
Contact: Haigang Sun    13823168465    sunhaigang@genomics.cn   
Sponsors and Collaborators
Shenzhen Hemogen
Investigators
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Principal Investigator: Chao Liu, PHD Shenzhen Hemogen
Principal Investigator: Sixi Liu, Professor Shenzhen Children's Hospital
More Information
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Responsible Party: Shenzhen Hemogen
ClinicalTrials.gov Identifier: NCT05745532    
Obsolete Identifiers: NCT04592458
Other Study ID Numbers: SZ-101
First Posted: February 27, 2023    Key Record Dates
Last Update Posted: February 27, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn