A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia (ET-01)

March 25, 2025 updated by: EdiGene (GuangZhou) Inc.

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

8

Phase

  • Phase 1

Contacts and Locations

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

Study Locations

  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Nanfang Hospital of Southern Medical University
      • Guangzhou, Guangdong, China, 510623
        • Guangzhou Women and Children's Medical Center
      • Shenzhen, Guangdong, China, 518035
        • Shenzhen Children's Hospital
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Institute of Hematology & Blood Diseases 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

12 years to 35 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.
  • Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.
  • Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.
  • Lansky/Karnofsky score ≥ 70%.
  • Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.

Other protocol defined inclusion criteria may apply.

Key Exclusion Criteria:

  • Subjects with associated α-thalassemia.
  • Subjects with any clinically significant acute or uncontrolled infections.
  • History of uncontrolled epilepsy or other mental disorders.
  • Previous treatment with allogeneic bone marrow transplantation 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
Biological: ET-01
Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency and severity of collected AEs & SAEs
Time Frame: Within 24 months after ET-01 infusion
Within 24 months after ET-01 infusion
All-cause mortality
Time Frame: From signing of informed consent up to 24 months post-ET-01 infusion
From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality
Time Frame: From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count
Time Frame: Within 24 months after ET-01 infusion
Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells
Time Frame: Within 24 months after ET-01 infusion
Within 24 months after ET-01 infusion

Collaborators and Investigators

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

Sponsor

EdiGene (GuangZhou) Inc.

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)

August 17, 2021

Primary Completion (Actual)

January 30, 2024

Study Completion (Actual)

March 12, 2025

Study Registration Dates

First Submitted

June 4, 2021

First Submitted That Met QC Criteria

June 11, 2021

First Posted (Actual)

June 14, 2021

Study Record Updates

Last Update Posted (Actual)

March 30, 2025

Last Update Submitted That Met QC Criteria

March 25, 2025

Last Verified

March 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EDG-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

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.

Clinical Trials on Transfusion Dependent Beta-Thalassaemia

Clinical Trials on ET-01

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kazakhstan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe