Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
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ClinicalTrials.gov Identifier: NCT05745532 |
Recruitment Status :
Recruiting
First Posted : February 27, 2023
Last Update Posted : February 27, 2023
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Condition or disease | Intervention/treatment | Phase |
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β-thalassemia | Biological: β-globin restored autologous hematopoietic stem cells | Early Phase 1 |
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 |

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.
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Biological: β-globin restored autologous hematopoietic stem cells
β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q |
- 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
- Overall survival [ Time Frame: 0-24 months ]Number of patients alive through the whole trial will be record
- 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
- Replication competent lentivirus (RCL) [ Time Frame: 0-24 months ]The percentage of RCL should be negative in the 24 months after transplant
- 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.
- 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
- 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
- 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

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Ages Eligible for Study: | 8 Years to 16 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

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
Contact: Haigang Sun | 13823168465 | sunhaigang@genomics.cn |
China, Guangdong | |
Shenzhen Children's Hospital | Recruiting |
Shenzhen, Guangdong, China, 518083 | |
Contact: Haigang Sun 13823168465 sunhaigang@genomics.cn |
Principal Investigator: | Chao Liu, PHD | Shenzhen Hemogen | |
Principal Investigator: | Sixi Liu, Professor | Shenzhen Children's Hospital |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |