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A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
ET-01
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
1 other identifier
interventional
8
1 country
4
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Aug 2021
Typical duration for phase_1
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 4, 2021
CompletedFirst Posted
Study publicly available on registry
June 14, 2021
CompletedStudy Start
First participant enrolled
August 17, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
March 12, 2025
CompletedMarch 30, 2025
March 1, 2025
2.5 years
June 4, 2021
March 25, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (5)
Frequency and severity of collected AEs & SAEs
Within 24 months after ET-01 infusion
All-cause mortality
From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality
From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count
Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells
Within 24 months after ET-01 infusion
Study Arms (1)
ET-01
EXPERIMENTALInterventions
Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Nanfang Hospital of Southern Medical University
Guangzhou, Guangdong, 510515, China
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510623, China
Shenzhen Children's Hospital
Shenzhen, Guangdong, 518035, China
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 4, 2021
First Posted
June 14, 2021
Study Start
August 17, 2021
Primary Completion
January 30, 2024
Study Completion
March 12, 2025
Last Updated
March 30, 2025
Record last verified: 2025-03
Data Sharing
- IPD Sharing
- Will not share