Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Probability of Long-term Disease-free Survival (DFS)
研究概览
简要总结
This protocol using busulfan, cyclophosphamide and melphalan has been designed as conditioning therapy for patients receiving stem cell transplantation for acute leukemia or myelodysplastic syndrome (MDS). The hypothesis is that this new regimen will be well tolerated and will cure the patient.
详细描述
Subjects will be admitted to the bone marrow transplant unit and put in isolation to reduce exposure to infectious agents.
Prior to transplantation, they will receive BUSULFAN via the central venous line, four times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's cancer. As well, these drugs will destroy their immune system to help ensure the new stem cells take and grow after transplantation.
On the day of transplantation, umbilical cord blood from the donor will be transfused via venous line. These new cells will replace the subject's bone marrow.
After transplantation, the subjects will receive Cyclosporin A and either MMF or MTX
Isolation will be continued until adequate numbers of cells are present in the blood to fight infection. Subjects will be discharged from the hospital when medically ready. They will be expected to return for follow-up to the blood and marrow transplant clinic at specific dates as determined by physicians.
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 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have a diagnosis of acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and currently be in complete remission.
- •Patients must be either:
- •- <18 years of age who are at least 6 months after initial hematopoietic cell transplant (HCT),
- •- 19-35 years of age and at least 18 months after initial HCT, or
- •- <35 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy
- •Adequate major organ function including:
- •- Cardiac: ejection fraction > or = 45%
- •- Renal: creatinine clearance > or = 40 mL/min
- •- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- •- Karnofsky performance status > or = 70% or Lansky score > or = 50%
- •Women of child bearing age must be using adequate birth control and have a negative pregnancy test.
- •Written informed consent.
排除标准
- •Eligible for TBI containing preparative regimen.
- •Active uncontrolled infection within one week of HCT.
- •Pregnant or lactating females.
研究组 & 干预措施
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: Stem Cell Transplant (Procedure)
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: Busulfan (Drug)
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: Cyclophosphamide (Drug)
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: Melphalan (Drug)
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: G-CSF (Drug)
Treatment Arm
Patients treated with therapy plan consisting of Busulfan every 6 hours on days -7 through -4, Cyclophosphamide 60 mg/kg/day IV x 2 days, Melphalan 140 mg/m on day -1, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation on day 0.
干预措施: ATG (Drug)
结局指标
主要结局
Probability of Long-term Disease-free Survival (DFS)
时间窗: 1 year
Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.
次要结局
- Incidence Chronic Graft-versus-host Disease (GVHD)(1 year)
- Probability of Engraftment(1 year)
- Incidence of Acute Graft-versus-host Disease (GVHD)(100 days post-transplant)
- Incidence of Regimen-related Toxicity 100 Days Post Transplant(100 days post-transplant)
- Incidence of Relapse(1 year)