NCT05752123终止不适用
A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
研究概览
简要总结
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
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
- 6 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
- •6~35 years old, all gender;
- •Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
- •Eligible for autologous stem cell transplant;
- •Organs in good function.
- •Other protocol defined Inclusion criteria may apply.
排除标准
- •Subjects with associated α-thalassemia;
- •Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
- •HLA identical sibling or unrelated donors are available;
- •Prior allo-HSCT or gene therapy.
- •Other protocol defined Exclusion criteria may apply.
研究组 & 干预措施
ET-01
Experimental
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells
干预措施: ET-01 (Biological)
结局指标
主要结局
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
时间窗: From ET-01 infusion to 104 weeks post-transplant
Incidence of transplant-related mortality.
时间窗: within 100 days post-transplant
All-cause mortality.
时间窗: From signing of informed consent to 104 weeks post-transplant
Proportion of subjects with engraftment.
时间窗: up to 42 days post-transplant
次要结局
- Change of proportion of HbF/Hb.(within 104 weeks post-transplant)
- Change of HbF from baseline.(within 104 weeks post-transplant)
- Change in total hemoglobin from baseline.(within 104 weeks post-transplant)
- Change of frequency of packed RBC transfusions.(From 6 months before recruitment to 104 weeks post-transplant)
- Change of volume of packed RBC transfusions.(From 6 months before recruitment to 104 weeks post-transplant)
研究者
研究点 (1)
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