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
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 4
- 主要终点
- Frequency and severity of collected AEs & SAEs
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
ET-01
干预措施: ET-01 (Biological)
结局指标
主要结局
Frequency and severity of collected AEs & SAEs
时间窗: Within 24 months after ET-01 infusion
All-cause mortality
时间窗: From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality
时间窗: From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count
时间窗: Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells
时间窗: Within 24 months after ET-01 infusion
次要结局
未报告次要终点