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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 11, 2023
Sponsor:
Collaborators:
EdiGene Inc.
The Affiliated Hospital Of Guizhou Medical University
Zunyi Medical College
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, multi-center 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 the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia
Actual Study Start Date : February 10, 2023
Estimated Primary Completion Date : June 15, 2025
Estimated Study Completion Date : August 15, 2025

Resource links provided by the National Library of Medicine


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. All-cause mortality [ Time Frame: From signing of informed consent to 104 weeks post-transplant ]
  3. Incidence of transplant-related mortality [ Time Frame: Within 100 days post-transplant ]
  4. Proportion of subjects with engraftment [ Time Frame: Up to 42 days post-transplant ]

Secondary Outcome Measures :
  1. Change of total hemoglobin from baseline [ Time Frame: Within 104 weeks post-transplant ]
  2. Change of HbF from baseline [ Time Frame: Within 104 weeks post-transplant ]
  3. Change of proportion of HbF/Hb [ Time Frame: Within 104 weeks post-transplant ]
  4. Change of frequency of packed RBC transfusions [ Time Frame: From 6 months before recruitment to 104 weeks post-transplant ]
  5. Change of 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;
  • Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
  • Eligible for autologous stem cell transplant;
  • Eligible for autologous stem cell transplant;
  • Organs in good function.
  • Other protocol defined inclusion criteria may apply.

Exclusion Criteria:

  • Subjects with associated α-thalassemia;
  • 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, Guizhou
The Affiliated Hospital of Guizhou Medical University Recruiting
Guiyang, Guizhou, China, 550004
Contact: Jiao Jin, MD    (0851)86777812    jinjiao999@163.com   
Affiliated Hospital of Zunyi Medical University Recruiting
Zunyi, Guizhou, China, 563000
Contact: Yan Chen, MD    (0851)28609207    cyz600@163.com   
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.
The Affiliated Hospital Of Guizhou Medical University
Zunyi Medical College
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 11, 2023
Last Verified: May 2023
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