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

• ClinicalTrials.gov Identifier: NCT04390971
• Recruitment Status: Active, not recruiting
• First Posted: May 18, 2020
• Last Update Posted: December 13, 2021
• 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

• First Submitted Date: May 12, 2020
• First Posted Date: May 18, 2020
• Last Update Posted Date: December 13, 2021
• Actual Study Start Date: August 11, 2020
• Estimated Primary Completion Date: April 30, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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 (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

Current Secondary Outcome Measures (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 (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: 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
• 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: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Transfusion Dependent Beta-Thalassaemia

Intervention

Biological: ET-01

Recruited participants will receive ET-01 IV infusion after conditioning.

Study Arms

Experimental: ET-01

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Intervention: Biological: ET-01

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: May 7, 2021): 1
• Original Estimated Enrollment (submitted: May 12, 2020): 3
• Estimated Study Completion Date: October 15, 2023
• Estimated Primary Completion Date: April 30, 2023 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years to 35 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: China

Administrative Information

• NCT Number: NCT04390971
• Other Study ID Numbers: 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

• Plan to Share IPD: No

• Current Responsible Party: Jun Shi, Institute of Hematology & Blood Diseases Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: Institute of Hematology & Blood Diseases Hospital
• Original Study Sponsor: Same as current
• Collaborators: EdiGene Inc.

Investigators

• Principal Investigator: Jun Shi, PhD; Institute of Hematology & Blood Diseases Hospital

• PRS Account: Institute of Hematology & Blood Diseases Hospital
• Verification Date: December 2021