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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 : May 10, 2021
Sponsor:
Collaborator:
EdiGene Inc.
Information provided by (Responsible Party):
Jun Shi, Institute of Hematology & Blood Diseases Hospital

Tracking Information
First Submitted Date  ICMJE May 12, 2020
First Posted Date  ICMJE May 18, 2020
Last Update Posted Date May 10, 2021
Actual Study Start Date  ICMJE August 11, 2020
Estimated Primary Completion Date October 31, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 26, 2020)
  • 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 12, 2020)
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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 26, 2020)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 12, 2020)
  • Engraftment of ET-01 in subjects with Transfusion Dependent β-Thalassaemia. [ Time Frame: within 42 days post-transplant ]
  • Effect on content and ratio of total hemoglobin, HbF and HbA of ET-01 transplantation. [ Time Frame: within 2 years post transplant ]
  • Effect on frequency and volume of packed RBC transfusions of ET-01 transplantation. [ Time Frame: within 2 years post transplant ]
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
Condition  ICMJE Transfusion Dependent Beta-Thalassaemia
Intervention  ICMJE Biological: ET-01
Recruited participants will receive ET-01 IV infusion after conditioning.
Study Arms  ICMJE Experimental: ET-01
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.
Intervention: Biological: ET-01
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: May 7, 2021)
1
Original Estimated Enrollment  ICMJE
 (submitted: May 12, 2020)
3
Estimated Study Completion Date  ICMJE October 15, 2023
Estimated Primary Completion Date October 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
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
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Jun Shi, Institute of Hematology & Blood Diseases Hospital
Study Sponsor  ICMJE Institute of Hematology & Blood Diseases Hospital
Collaborators  ICMJE EdiGene Inc.
Investigators  ICMJE
Principal Investigator: Jun Shi, PhD Institute of Hematology & Blood Diseases Hospital
PRS Account Institute of Hematology & Blood Diseases Hospital
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP