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Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

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 28, 2020
Sponsor:
Collaborator:
EdiGene Inc.
Information provided by (Responsible Party):
Jun Shi, Institute of Hematology & Blood Diseases Hospital

Study Description
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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
Transfusion Dependent Beta-Thalassaemia Biological: ET-01 Not Applicable

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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 21, 2020
Estimated Primary Completion Date : October 31, 2021
Estimated Study Completion Date : October 15, 2023

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Arms and Interventions
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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
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Primary Outcome Measures :
  1. Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0. [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
  2. Incidence of abnormal laboratory test results after transplant [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
  3. Incidence of abnormal vital signs after transplant [ Time Frame: from ET-01 infusion to 104 weeks post-transplant ]
  4. Incidence of clinical significant abnormal vital signs. [ Time Frame: from ET-01 infusion to 60mins after ET-01 infusion ]
  5. Proportion of subjects with clinical significant abnormal vital signs. [ Time Frame: from ET-01 infusion to 60mins after ET-01 infusion ]
  6. Mortality after ET-01 infusion. [ Time Frame: within 100 days post-transplant ]

Secondary Outcome Measures :
  1. 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

  2. Change in total hemoglobin from baseline. [ Time Frame: within 104 weeks post-transplant ]
  3. Change in HbF from baseline. [ Time Frame: within 104 weeks post-transplant ]
  4. Change in HbA from baseline. [ Time Frame: within 104 weeks post-transplant ]
  5. Change in proportion of HbF/HbA. [ Time Frame: within 104 weeks post-transplant ]
  6. Change in frequency of packed RBC transfusions. [ Time Frame: from 6 months before recruitment to 104 weeks post-transplant ]
  7. Change in volume of packed RBC transfusions. [ Time Frame: from 6 months before recruitment to 104 weeks post-transplant ]

Eligibility Criteria
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Ages Eligible for Study:   6 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Jun Shi, PhD (86)2223900913 shijun@ihcams.ac.cn
Contact: Jingyu Zhao, MPH (86)13752253515 zhaojingyu@ihcams.ac.cn

Locations
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China, Tianjin
Institute of Hematology & Blood Diseases Hospital Recruiting
Tianjin, Tianjin, China, 300020
Contact: Jun Shi         
Sponsors and Collaborators
Institute of Hematology & Blood Diseases Hospital
EdiGene Inc.
Investigators
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Principal Investigator: Jun Shi, PhD Institute of Hematology & Blood Diseases Hospital
More Information
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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 28, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jun Shi, Institute of Hematology & Blood Diseases Hospital:
Beta-Thalassaemia
Hematopoietic Stem-Cell Transplantation
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn