Back to resultsA Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (ET-01)
Primary Purpose
Transfusion Dependent Beta-Thalassaemia
Status
Active
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
ET-01
Sponsored by
About this trial
This is an interventional treatment trial for Transfusion Dependent Beta-Thalassaemia focused on measuring Beta-Thalassaemia, CRISPR-Cas9, Human Hematopoietic Stem and Progenitor Cells
Eligibility Criteria
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.
Sites / Locations
- Nanfang Hospital of Southern Medical University
- Guangzhou Women and Children's Medical Center
- Shenzhen Children's Hospital
- Institute of Hematology & Blood Diseases Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ET-01
Arm Description
Outcomes
Primary Outcome Measures
Frequency and severity of collected AEs & SAEs
All-cause mortality
Incidence of transplant-related mortality
Total lymphocyte count
Proportion of subjects with abnormal proliferation of blood cells
Secondary Outcome Measures
Full Information
NCT ID
NCT04925206
First Posted
June 4, 2021
Last Updated
February 15, 2023
Sponsor
EdiGene (GuangZhou) Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04925206
Brief Title
A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia
Acronym
ET-01
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 17, 2021 (Actual)
Primary Completion Date
June 30, 2024 (Anticipated)
Study Completion Date
June 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
EdiGene (GuangZhou) Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transfusion Dependent Beta-Thalassaemia
Keywords
Beta-Thalassaemia, CRISPR-Cas9, Human Hematopoietic Stem and Progenitor Cells
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ET-01
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
ET-01
Intervention Description
Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.
Primary Outcome Measure Information:
Title
Frequency and severity of collected AEs & SAEs
Time Frame
Within 24 months after ET-01 infusion
Title
All-cause mortality
Time Frame
From signing of informed consent up to 24 months post-ET-01 infusion
Title
Incidence of transplant-related mortality
Time Frame
From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Title
Total lymphocyte count
Time Frame
Within 24 months after ET-01 infusion
Title
Proportion of subjects with abnormal proliferation of blood cells
Time Frame
Within 24 months after ET-01 infusion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Nanfang Hospital of Southern Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Facility Name
Guangzhou Women and Children's Medical Center
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510623
Country
China
Facility Name
Shenzhen Children's Hospital
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518035
Country
China
Facility Name
Institute of Hematology & Blood Diseases Hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia
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