NCT02014506Unknown1 期
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft
研究概览
简要总结
Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.
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
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A. Disease inclusions
- •Hematologic malignancy:
- •Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features
- •Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome
- •Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion
- •Chronic myeloid leukemia in AP
- •Juvenile myelomonocytic leukemia
- •Malignant lymphoma, NHL or HD, after failed autologous HSCT
- •Non-hematologic malignancy
- •Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on
- •Non-malignant hematologic disease
- •Acquired severe and very severe aplastic anemia
- •Fanconi anemia
- •Paroxysmal nocturnal hemoglobinuria
- •Congenital dyserythropoietic anemia
- •Inherited or metabolic disease
- •Hemophagocytic lymphohistiocytosis
- •Malignant osteopetrosis
- •Storage diseases
- •Others B. Recipient inclusions
- •Age < 21 years
- •No HLA-identical stem cell donor available
- •Lansky-Play performance score >60
- •No active infection at the time of transplantation
排除标准
- •HIV-infection
- •Presence of active and serious infection
- •Cardiac ejection fraction <35% on echocardiography
- •Severe pulmonary dysfunction (DLCO <30%)
- •Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L
- •Concurrent severe or uncontrolled medical disease
- •Patients who are pregnant
- •Patients unwilling or unable to comply with the protocol or unable to give informed consent
研究组 & 干预措施
HAPLO
Experimental
干预措施: Fludarabine (Drug)
HAPLO
Experimental
干预措施: Cyclophosphamide (Drug)
HAPLO
Experimental
干预措施: anti-thymocyte globulin (Biological)
HAPLO
Experimental
干预措施: filgrastim (Biological)
HAPLO
Experimental
干预措施: Total body irradiation (Radiation)
HAPLO
Experimental
干预措施: TCRαβ-depleted hematopoietic cell transplantation (Procedure)
HAPLO
Experimental
干预措施: CliniMACS (Device)
结局指标
主要结局
To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft
时间窗: 1 year posttransplant
次要结局
- To estimate the reactivation rate of CMV, EBV(100 days and 1 year posttransplant)
- To estimate the incidence of relapse(100 days and 1 year post-transplant)
- To estimate the incidence and severity of chronic GVHD(1 year posttransplant)
- To estimate the incidence of bacterial, fungal and viral infection(100 days and 1 year posttransplant)
- To evaluate the immune reconstitution of T, B, and NK cells(days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant)
- To evaluate the lineage-specific chimerism using flow cytomery of CD3+, CD19, CD56, TCR αβ, and TCRγδ at pre-transplant(days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant)
- To assess event free survival(1 year posttransplant)
- To assess engraftment and graft failure(28 days posttransplant)
- To estimate the risk of acute GVHD(100 days posttransplant)
- To estimate the overall survival(1 year posttransplant)
研究者
Ho Joon Im
Professor
Asan Medical Center
研究点 (1)
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