A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 3
- 主要终点
- Incidence of transplant-related mortality
研究概览
简要总结
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
详细描述
After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.
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;
- •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
BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.
干预措施: ET-01 (Biological)
结局指标
主要结局
Incidence of transplant-related mortality
时间窗: Within 100 days post-transplant
Proportion of subjects with engraftment
时间窗: Up to 42 days post-transplant
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
All-cause mortality
时间窗: From signing of informed consent to 104 weeks post-transplant
次要结局
- Change of proportion of HbF/Hb(Within 104 weeks post-transplant)
- Change of total hemoglobin from baseline(Within 104 weeks post-transplant)
- Change of HbF 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
Jun Shi
Director of Regenerative Medicine Clinic Center
Institute of Hematology & Blood Diseases Hospital, China