A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia (NCT04925206) | Clinical Trial Compass
TerminatedPhase 1
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
Stopped: Development plan change
China8 participantsStarted 2021-08-17
Plain-language 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.
Who can participate
Age range
12 Years – 35 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Frequency and severity of collected AEs & SAEs
Timeframe: Within 24 months after ET-01 infusion
2
All-cause mortality
Timeframe: From signing of informed consent up to 24 months post-ET-01 infusion
3
Incidence of transplant-related mortality
Timeframe: From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
4
Total lymphocyte count
Timeframe: Within 24 months after ET-01 infusion
5
Proportion of subjects with abnormal proliferation of blood cells