Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 98
- 试验地点
- 2
- 主要终点
- Overall Survival
研究概览
简要总结
Treatment Study to assess of safety and efficiency of T cells receptor (TCR) alfa beta depleted graft for hematopoietic stem cell transplantation (HSCT) from haploidentical and unrelated donors in patients with primary immunodeficiency diseases
详细描述
Infections, graft versus host diseases (GVHD) and associated morbidity and mortality remains significant problems after unrelated and haploidentical hematopoietic stem sell transplantation (HSCT) in patients with primary immunodeficiency diseases (PID). In this study the hypothesis is that the transplantation of TCR alfa beta depleted peripheral blood stem cells (PBSC) would offers advantages over the use of positively selected CD34+ stem cells in haploidentical HSCT and non-manipulated graft in unrelated HSCT.
The purpose of this study is to evaluate the safety and efficiency of the selective infusion of TCR alfa beta T cell depleted graft in pediatric patients with PID receiving HSCT from haploidentical and unrelated donors.
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
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥ 1 months and < 19 years
- •Patients diagnosed with Primary Immunodeficiency Diseases eligible for an allogeneic transplantation and lacking a related HLA-matched donor
- •Lansky/Karnofsky score > 40, WHO > 4
- •Signed written informed consent
排除标准
- •Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- •Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- •Serious concurrent uncontrolled medical disorder
- •Pregnant or breast feeding female patient
- •Lack of parents' informed consent.
结局指标
主要结局
Overall Survival
时间窗: 1 year after HSCT
The probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT
次要结局
- Percentage of Patients With Full Donor Chimerism(last follow-up)
- Acute Graft Versus Host Diseases (аGVHD)(12 months after transplantation)
- Viral Infections After Transplant(12 months after transplantation)
- Transplant Related Mortality (TRM)(24 months after transplantation)
- Chronic Graft Versus Host Diseases (cGVHD)(1 year after HSCT)
- Cellular Immunological Reconstitution(2 years after HSCT)