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History of Changes for Study: NCT04390971
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
Latest version (submitted December 9, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 May 12, 2020 None (earliest Version on record)
2 May 26, 2020 Recruitment Status, Outcome Measures, Study Status, Arms and Interventions, Contacts/Locations, Study Description, Oversight and Sponsor/Collaborators
3 September 8, 2020 Study Status
4 November 22, 2020 Study Status
5 February 20, 2021 Study Status
6 May 7, 2021 Recruitment Status, Study Status, Contacts/Locations and Study Design
7 December 9, 2021 Study Status
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Study NCT04390971
Submitted Date:  May 12, 2020 (v1)

Open or close this module Study Identification
Unique Protocol ID: EDI-001
Brief Title: Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
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
Secondary IDs:
Open or close this module Study Status
Record Verification: May 2020
Overall Status: Not yet recruiting
Study Start: June 1, 2020
Primary Completion: October 31, 2021 [Anticipated]
Study Completion: October 15, 2023 [Anticipated]
First Submitted: May 12, 2020
First Submitted that
Met QC Criteria:
May 12, 2020
First Posted: May 18, 2020 [Actual]
Last Update Submitted that
Met QC Criteria:
May 12, 2020
Last Update Posted: May 18, 2020 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Institute of Hematology & Blood Diseases Hospital
Responsible Party: Principal Investigator
Investigator: Jun Shi
Official Title: Director of Regenerative Medicine Clinic Center
Affiliation: Institute of Hematology & Blood Diseases Hospital
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: No
U.S. FDA-regulated Device: No
Data Monitoring: No
Open or close this module 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.
Detailed Description: After proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 transfusion to complete therapy. 2 years' follow up visits will be carried out post-transplantation and related data will be collected.
Open or close this module Conditions
Conditions: Transfusion Dependent Beta-Thalassaemia
Keywords: Beta-Thalassaemia
Hematopoietic Stem-Cell Transplantation
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Not Applicable
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 3 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: ET-01
Each recruited subject will accept ET-01 Transplantation.
Biological: ET-01
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 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
Secondary Outcome Measures:
1. Engraftment of ET-01 in subjects with Transfusion Dependent β-Thalassaemia.
[ Time Frame: within 42 days post transplant ]

2. Effect on content and ratio of total hemoglobin, HbF and HbA of ET-01 transplantation.
[ Time Frame: within 2 years post transplant ]

3. Effect on frequency and volume of packed RBC transfusions of ET-01 transplantation.
[ Time Frame: within 2 years post transplant ]

Open or close this module Eligibility
Minimum Age: 6 Years
Maximum Age: 35 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

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.
Open or close this module Contacts/Locations
Central Contact Person: Jun Shi, PhD
Telephone: (86)2223900913
Email: shijun@ihcams.ac.cn
Central Contact Backup: Jingyu Zhao, MPH
Telephone: (86)13752253515
Email: zhaojingyu@ihcams.ac.cn
Study Officials: Jun Shi, PhD
Principal Investigator
Institute of Hematology & Blood Diseases Hospital
Locations:
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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