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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Sponsor:
Institute of Hematology & Blood Diseases Hospital
Collaborator:
EdiGene Inc.
Information provided by (Responsible Party):
Jun Shi, Institute of Hematology & Blood Diseases Hospital
Tracking Information | |||||
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First Submitted Date ICMJE | May 12, 2020 | ||||
First Posted Date ICMJE | May 18, 2020 | ||||
Last Update Posted Date | December 13, 2021 | ||||
Actual Study Start Date ICMJE | August 11, 2020 | ||||
Estimated Primary Completion Date | April 30, 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
Safety of ET-01 single transplantation in subjects with Transfusion Dependent β-Thalassaemia [ Time Frame: within 2 years post transplant ] Incidence of AEs and SAEs post transplant
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia | ||||
Official Title ICMJE | A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia | ||||
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. | ||||
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 Type ICMJE | Interventional | ||||
Study Phase ICMJE | Not Applicable | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Transfusion Dependent Beta-Thalassaemia | ||||
Intervention ICMJE | Biological: ET-01
Recruited participants will receive ET-01 IV infusion after conditioning.
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Study Arms ICMJE | Experimental: ET-01
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.
Intervention: Biological: ET-01
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
1 | ||||
Original Estimated Enrollment ICMJE |
3 | ||||
Estimated Study Completion Date ICMJE | October 15, 2023 | ||||
Estimated Primary Completion Date | April 30, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years to 35 Years (Child, Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | China | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT04390971 | ||||
Other Study ID Numbers ICMJE | EDI-001 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Jun Shi, Institute of Hematology & Blood Diseases Hospital | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Institute of Hematology & Blood Diseases Hospital | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | EdiGene Inc. | ||||
Investigators ICMJE |
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PRS Account | Institute of Hematology & Blood Diseases Hospital | ||||
Verification Date | December 2021 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |