A Phase II Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children and Young Adults With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Number of Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
研究概览
简要总结
The purpose of this study is to compare safety and efficacy of reduced-intensity conditioning and myeloablative conditioning regimens prior to HSCT in high-risk AML/MDS pediatric and young adult patients. This study investigates the use of two novel conditioning therapies for hematopoietic stem cell transplant (HSCT). The primary focus of both the investigators' myeloablative and reduced-intensity conditioning regimens is to reduce overall toxicity so that pediatric and young adult patients with high-risk AML/MDS with significant pretransplant comorbidities who would have been ineligible to proceed to HSCT previously can now receive potentially life-saving treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Months 至 26 Years（Child, 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
Reduced-Intensity Conditioning
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
干预措施: Reduced-Intensity Conditioning Regimen (Drug)
Myeloablative Conditioning
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) , Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
干预措施: Myeloablative Conditioning Regimen (Drug)
结局指标
主要结局
Number of Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
时间窗: Day 100
Number of non-relapsed deaths that occur
Number of Non-relapsed Deaths by 6 Months Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
时间窗: Day 180
Number of non-relapsed deaths that occur
Preliminary Efficacy (Event-free Survival at 6 Months) in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
时间窗: 6 months
Event-free survival at 6 months, where events are defined as relapse or death
次要结局
- The Pace of Platelet Recovery(Day of transplant to end of study (Day 365))
- Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade(Day of transplant to end of study (Day 365))
- Day 0 Campath (Alemtuzumab) Level(Day 0)
- The Number of Subjects With Overall Survival (OS)(Day 100 and 180 post-transplant)
- Pace of Immune Reconstitution(Day 180)
- The Pace of Neutrophil Recovery(Day of transplant to end of study (Day 365))
- Incidence of Primary Graft Failure.(Post-transplant to 42 days post-transplant)
- Incidence of Grades 4 and 5 Adverse Events(Day 180)
- Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade(Day of transplant to end of study (Day 365))
- Disease-free Survival (DFS)(Day 100 and 180 post-transplant)
- Treatment-related Mortality (TRM)(Day 100 and 180 post-transplant)
研究者
Randy Windreich
Assistant Professor of Medicine
University of Pittsburgh