NCT04925206TerminatedPhase 1
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
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
Source: ClinicalTrials.gov · Last updated 30 Mar 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 8 (actual)
- First submitted
- 4 Jun 2021
- Start date
- 17 Aug 2021
- Primary completion date
- 30 Jan 2024
- Lead sponsor
- EdiGene (GuangZhou) Inc.
- Diseases
- Thalassaemia
- NCT number
- NCT04925206
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment with allogeneic bone marrow transplantation or gene therapy.
- Other protocol defined exclusion criteria may apply.
Other illness
- Subjects with any clinically significant acute or uncontrolled infections.
- History of uncontrolled epilepsy or other mental disorders.
Other
- Subjects with associated α-thalassemia.
Who the study is looking for
- 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.
Age in the registry: 12 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (4)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Nanfang Hospital of Southern Medical University Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Guangzhou Women and Children's medical center Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Shenzhen Children's Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
| Institute of Hematology & Blood Diseases Hospital Coordinator of this site? Confirm its status | Tianjin | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.