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

NCT04390971 | Completed

• 1 other identifier: SC2019004
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 3

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.

Conditions

Transfusion Dependent Beta-Thalassaemia

Keywords

Beta-Thalassaemia; Hematopoietic Stem-Cell Transplantation

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (5)

• 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

Study Arms (1)

ET-01

EXPERIMENTAL

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Biological: ET-01

Interventions

ET-01 BIOLOGICAL

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

ET-01

Eligibility Criteria

• Age: 6 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
• \~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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• EdiGene Inc.: collaborator
• The Affiliated Hospital Of Guizhou Medical University: collaborator
• Zunyi Medical College: collaborator

Study Sites (3)

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, 550004, China

Location

Affiliated Hospital of Zunyi Medical University

Zunyi, Guizhou, 563000, China

Location

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jun Shi, PhD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2020
• First Posted: May 18, 2020
• Study Start: February 10, 2023
• Primary Completion: March 5, 2023
• Study Completion: March 5, 2023
• Last Updated: February 11, 2026

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)