A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.

June 6, 2023 updated by: EdiGene (GuangZhou) Inc.

A Single Site, 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, single site 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

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

2

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Guangxi
      • Nanning, Guangxi, China, 530021
        • PLA 923 Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 35 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Key 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;
  • Organs in good function.

Other protocol defined Inclusion criteria may apply.

Key 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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.
Time Frame: From ET-01 infusion to 104 weeks post-transplant
From ET-01 infusion to 104 weeks post-transplant
All-cause mortality.
Time Frame: From signing of informed consent to 104 weeks post-transplant
From signing of informed consent to 104 weeks post-transplant
Incidence of transplant-related mortality.
Time Frame: within 100 days post-transplant
within 100 days post-transplant
Proportion of subjects with engraftment.
Time Frame: up to 42 days post-transplant
up to 42 days post-transplant

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

EdiGene (GuangZhou) Inc.

Collaborators

PLA 923 Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 18, 2023

Primary Completion (Estimated)

June 15, 2025

Study Completion (Estimated)

August 15, 2025

Study Registration Dates

First Submitted

February 16, 2023

First Submitted That Met QC Criteria

February 21, 2023

First Posted (Actual)

March 2, 2023

Study Record Updates

Last Update Posted (Actual)

June 8, 2023

Last Update Submitted That Met QC Criteria

June 6, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EDI-001-002

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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