Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
Study Details
Study Description
Brief Summary
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
N/A |
Detailed Description
After proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. 2 years' follow up visits will be carried out post-transplantation and related data will be collected.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ET-01 BCL11A Enhancer modified Autologous Hematopoietic Stem Cells. |
Biological: ET-01
Recruited participants will receive ET-01 IV infusion after conditioning.
|
Outcome Measures
Primary Outcome Measures
- Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0. [from ET-01 infusion to 104 weeks post-transplant]
- Incidence of abnormal laboratory test results after transplant [from ET-01 infusion to 104 weeks post-transplant]
- Incidence of abnormal vital signs after transplant [from ET-01 infusion to 104 weeks post-transplant]
- Incidence of clinical significant abnormal vital signs. [from ET-01 infusion to 60mins after ET-01 infusion]
- Proportion of subjects with clinical significant abnormal vital signs. [from ET-01 infusion to 60mins after ET-01 infusion]
- Mortality after ET-01 infusion. [within 100 days post-transplant]
Secondary Outcome Measures
- Engraftment of ET-01 in subjects. [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. [within 104 weeks post-transplant]
- Change in HbF from baseline. [within 104 weeks post-transplant]
- Change in HbA from baseline. [within 104 weeks post-transplant]
- Change in proportion of HbF/HbA. [within 104 weeks post-transplant]
- Change in frequency of packed RBC transfusions. [from 6 months before recruitment to 104 weeks post-transplant]
- Change in volume of packed RBC transfusions. [from 6 months before recruitment to 104 weeks post-transplant]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
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6~35 years old, all gender, weight ≥ 30kg;
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Genetically diagnosed β-Thalassaemia;
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Diagnosed as transfusion-dependent;
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Eligible for autologous stem cell transplant;
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Organs in good function;
-
Other protocol defined Inclusion criteria may apply.
Exclusion Criteria:
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Thalassemia gene type is β0/β0;
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Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
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HLA identical sibling or unrelated donors are available;
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Prior allo-HSCT or gene therapy;
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Other protocol defined Exclusion criteria may apply.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Institute of Hematology & Blood Diseases Hospital | Tianjin | Tianjin | China | 300020 |
Sponsors and Collaborators
- Institute of Hematology & Blood Diseases Hospital
- EdiGene Inc.
Investigators
- Principal Investigator: Jun Shi, PhD, Institute of Hematology & Blood Diseases Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- EDI-001