A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Proportion of saGVHD Using a ProphylaxisRegimen With no Calcineurin Inhibitors After Day 100 Post First Allogeneic HLA-Matched Sibling or Unrelated Donor HCT for Hematological Malignancies.
研究概览
简要总结
The participants are being asked to take part in this clinical trial because the participant have a lymphoid or myeloid based cancer diagnosis that requires a bone marrow transplant.
Primary Objectives
To estimate the incidence of severe acute GVHD (saGVHD) using a prophylaxis regimen with no calcineurin inhibitors after day +60 post first allogeneic Human Leukocyte antigen (HLA)-matched sibling or unrelated donor HCT for hematological malignancies.
Secondary objective
Determine the cumulative incidence of relapse, NRM, chronic GVHD, and OS in study participants at one year post-transplant.
Exploratory objectives
- To evaluate the pharmacokinetic/pharmacodynamic (PK/PD) profiles of ruxolitinib, fludarabine, and rATG.
- To assess immune reconstitution in study participants within the first year post-HCT.
详细描述
The investigator propose to employ two preparative regimens based on the underlying hematological malignancy. For hematological malignancies of the lymphoid lineage we will use a standard preparative regimen consisting of Total Body Irradiation and cyclophosphamide (TBI/Cy), unless TBI is contraindicated. For myeloid malignancies we will use thiotepa, busulfan, and fludarabine (TBF), a preparative regimen that has been associated with a reduced risk of relapse and trend for improved survival with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy), our current regimen. All HCT recipients will receive cyclosporine in combination with methotrexate and ruxolitinib as GVHD prophylaxis. Recipients of MUD HCT will receive rATG for additional immunosuppression as is standard for unrelated donor transplants
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Transplant Patients
干预措施: Cyclophosphamide (Drug)
Transplant Patients
干预措施: Ruxolitinib (Drug)
Transplant Patients
干预措施: Mesna (Drug)
Transplant Patients
干预措施: Anti-thymocyte globulin (ATG) (Drug)
Transplant Patients
干预措施: Cyclosporine (Drug)
Transplant Patients
干预措施: Fludarabine (Drug)
Transplant Patients
干预措施: Methotrexate (Drug)
Transplant Patients
干预措施: Total Body Irradiation (radiation treatment) (Radiation)
Transplant Patients
干预措施: Bone marrow infusion (Drug)
Transplant Patients
干预措施: Busulfan (Drug)
Transplant Patients
干预措施: Thiotepa (Drug)
结局指标
主要结局
Proportion of saGVHD Using a ProphylaxisRegimen With no Calcineurin Inhibitors After Day 100 Post First Allogeneic HLA-Matched Sibling or Unrelated Donor HCT for Hematological Malignancies.
时间窗: 100 days post transplant
Development of Severe Acute GVHD (saGVHD) at or before Day 100 post transplant is considered as an event. Severe acute GVHD is defined as grade II-IVGVHD. Acute graft-vs-host disease will be evaluated using the standard grading criteria.
次要结局
- Cumulative Incidence of Overall Survival (OS)(One-year post-transplantation.)
- Cumulative Incidence of Relapse(One-year post-transplantation.)
- Cumulative Incidence of Non Relapse Mortality (NRM)(One-year post-transplantation.)
- Cumulative Incidence of Chronic GVHD(One-year post-transplantation.)