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: NCT04592458|
Recruitment Status : Not yet recruiting
First Posted : October 19, 2020
Last Update Posted : October 19, 2020
|Condition or disease||Intervention/treatment||Phase|
|β-Thalassemia Major||Biological: β-globin restored autologous HSC||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Study Start Date :||November 1, 2020|
|Estimated Primary Completion Date :||November 30, 2022|
|Estimated Study Completion Date :||November 30, 2024|
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.
Biological: β-globin restored autologous HSC
β-globin restored autologous HSC modified with lentiviral vector 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 engraftments [ 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 at 6, 12, 18 and 24 months after transplant. 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 and volume of packed RBC transfusions [ Time Frame: 18-24 Months ]Compare the annualized number of pRBC transfusions before gene therapy with the Month 6 and Month 24 period after transplant, the percentage change will be recorded.
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): NCT04592458
|Contact: Jing Li, PhDemail@example.com|
|Beijing Genomics Institute At Shenzhen|
|Shenzhen, Guangdong, China, 518083|
|Contact: Jing Li, PhD +8613510560664 firstname.lastname@example.org|
|Principal Investigator: Chao Liu, PhD|
|Principal Investigator: Sixi Liu, Professor|
|Principal Investigator:||Chao Liu, PhD||BGI-research|
|Principal Investigator:||Sixi Liu, Professor||Shenzhen Children's Hospital|