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

A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Transfusion Dependent Beta-Thalassaemia
• Intervention / Treatment: Biological: ET-01

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 Type: Interventional
• Enrollment (Actual): 3
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Jun Shi, PhD; Phone Number: (86)2223900913; Email: shijun@ihcams.ac.cn
• Study Contact Backup: Name: Jingyu Zhao, MPH; Phone Number: (86)13752253515; Email: zhaojingyu@ihcams.ac.cn

Study Locations

• China
  • Guizhou
    • Guiyang, Guizhou, China, 550004
      • The Affiliated Hospital Of GuiZhou Medical University
    • Zunyi, Guizhou, China, 563000
      • Affiliated Hospital of Zunyi Medical University
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Institute of Hematology & Blood Diseases Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 35 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.	From ET-01 infusion to 104 weeks post-transplant
All-cause mortality	From signing of informed consent to 104 weeks post-transplant
Incidence of transplant-related mortality	Within 100 days post-transplant
Proportion of subjects with engraftment	Up to 42 days post-transplant

Secondary Outcome Measures

Outcome Measure	Time Frame
Change of total hemoglobin from baseline	Within 104 weeks post-transplant
Change of HbF from baseline	Within 104 weeks post-transplant
Change of proportion of HbF/Hb	Within 104 weeks post-transplant
Change of frequency of packed RBC transfusions	From 6 months before recruitment to 104 weeks post-transplant
Change of volume of packed RBC transfusions	From 6 months before recruitment to 104 weeks post-transplant

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Collaborators: EdiGene Inc.; The Affiliated Hospital Of Guizhou Medical University; Zunyi Medical College
• Investigators: Principal Investigator: Jun Shi, PhD, Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2023
• Primary Completion (Estimated): June 15, 2025
• Study Completion (Estimated): August 15, 2025

Study Registration Dates

• First Submitted: May 12, 2020
• First Submitted That Met QC Criteria: May 12, 2020
• First Posted (Actual): May 18, 2020

Study Record Updates

• Last Update Posted (Actual): November 27, 2023
• Last Update Submitted That Met QC Criteria: November 23, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Beta-Thalassaemia; Hematopoietic Stem-Cell Transplantation
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: EDI-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No