Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
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ClinicalTrials.gov Identifier: NCT04390971 |
Recruitment Status :
Active, not recruiting
First Posted : May 18, 2020
Last Update Posted : December 13, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Transfusion Dependent Beta-Thalassaemia | Biological: ET-01 | Not Applicable |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 1 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia |
Actual Study Start Date : | August 11, 2020 |
Estimated Primary Completion Date : | April 30, 2023 |
Estimated Study Completion Date : | October 15, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: ET-01
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.
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Biological: ET-01
Recruited participants will receive ET-01 IV infusion after conditioning. |
- Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0. [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
- Incidence of abnormal laboratory test results after transplant [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
- Incidence of abnormal vital signs after transplant [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
- Incidence of clinical significant abnormal vital signs. [ Time Frame: from ET-01 infusion to 60mins after ET-01 infusion ]
- Proportion of subjects with clinical significant abnormal vital signs. [ Time Frame: from ET-01 infusion to 60mins after ET-01 infusion ]
- Mortality after ET-01 infusion. [ Time Frame: within 100 days post-transplant ]
- Engraftment of ET-01 in subjects. [ Time Frame: within 42 days post-transplant ]neutrophil count [ANC] >500 /mm3 for 3 consecutive days or platelet count [PLT] >20000/mm3 for 7 consecutive days
- Change in total hemoglobin from baseline. [ Time Frame: within 104 weeks post-transplant ]
- Change in HbF from baseline. [ Time Frame: within 104 weeks post-transplant ]
- Change in HbA from baseline. [ Time Frame: within 104 weeks post-transplant ]
- Change in proportion of HbF/HbA. [ Time Frame: within 104 weeks post-transplant ]
- Change in frequency of packed RBC transfusions. [ Time Frame: from 6 months before recruitment to 104 weeks post-transplant ]
- Change in volume of packed RBC transfusions. [ Time Frame: from 6 months before recruitment to 104 weeks post-transplant ]

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Ages Eligible for Study: | 6 Years to 35 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
- 6~35 years old, all gender, weight ≥ 30kg;
- Genetically diagnosed β-Thalassaemia;
- Diagnosed as transfusion-dependent;
- Eligible for autologous stem cell transplant;
- Organs in good function;
- Other protocol defined Inclusion criteria may apply.
Exclusion Criteria:
- Thalassemia gene type is β0/β0;
- Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
- HLA identical sibling or unrelated donors are available;
- Prior allo-HSCT or gene therapy;
- Other protocol defined Exclusion criteria may apply.

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): NCT04390971
China, Tianjin | |
Institute of Hematology & Blood Diseases Hospital | |
Tianjin, Tianjin, China, 300020 |
Principal Investigator: | Jun Shi, PhD | Institute of Hematology & Blood Diseases Hospital |
Responsible Party: | Jun Shi, Director of Regenerative Medicine Clinic Center, Institute of Hematology & Blood Diseases Hospital |
ClinicalTrials.gov Identifier: | NCT04390971 |
Other Study ID Numbers: |
EDI-001 |
First Posted: | May 18, 2020 Key Record Dates |
Last Update Posted: | December 13, 2021 |
Last Verified: | December 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Beta-Thalassaemia Hematopoietic Stem-Cell Transplantation |
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |