Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
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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: NCT04390971 |
Recruitment Status :
Recruiting
First Posted : May 18, 2020
Last Update Posted : May 11, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Transfusion Dependent Beta-Thalassaemia | Biological: ET-01 | Not Applicable |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 6 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia |
Actual Study Start Date : | February 10, 2023 |
Estimated Primary Completion Date : | June 15, 2025 |
Estimated Study Completion Date : | August 15, 2025 |

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 ]
- All-cause mortality [ Time Frame: From signing of informed consent to 104 weeks post-transplant ]
- Incidence of transplant-related mortality [ Time Frame: Within 100 days post-transplant ]
- Proportion of subjects with engraftment [ Time Frame: Up to 42 days post-transplant ]
- Change of total hemoglobin from baseline [ Time Frame: Within 104 weeks post-transplant ]
- Change of HbF from baseline [ Time Frame: Within 104 weeks post-transplant ]
- Change of proportion of HbF/Hb [ Time Frame: Within 104 weeks post-transplant ]
- Change of frequency of packed RBC transfusions [ Time Frame: From 6 months before recruitment to 104 weeks post-transplant ]
- Change of 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;
- Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
- Eligible for autologous stem cell transplant;
- Eligible for autologous stem cell transplant;
- Organs in good function.
- Other protocol defined inclusion criteria may apply.
Exclusion Criteria:
- Subjects with associated α-thalassemia;
- 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
Contact: Jun Shi, PhD | (86)2223900913 | shijun@ihcams.ac.cn | |
Contact: Jingyu Zhao, MPH | (86)13752253515 | zhaojingyu@ihcams.ac.cn |
China, Guizhou | |
The Affiliated Hospital of Guizhou Medical University | Recruiting |
Guiyang, Guizhou, China, 550004 | |
Contact: Jiao Jin, MD (0851)86777812 jinjiao999@163.com | |
Affiliated Hospital of Zunyi Medical University | Recruiting |
Zunyi, Guizhou, China, 563000 | |
Contact: Yan Chen, MD (0851)28609207 cyz600@163.com | |
China, Tianjin | |
Institute of Hematology & Blood Diseases Hospital | Recruiting |
Tianjin, Tianjin, China, 300020 | |
Contact: Jun Shi |
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: | May 11, 2023 |
Last Verified: | May 2023 |
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 |