A Single Arm Phase II Trial of Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 7
- 主要终点
- Relapse Rate
研究概览
简要总结
The purpose of this study is to learn if 5'-Azacitidine will help to lower the risk of the disease coming back after a stem cell transplant in patients with MDS and AML. This study will also be looking at the side effects of this medicine.
5'-Azacitidine is an FDA approved drug for treatment of MDS and AML, as well as patients whose disease came back after transplant, where it helped going into remission. It is unclear if 5'-Azacitidine can prevent the disease from coming back after transplant. This study will help show if getting 5'-Azacitidine soon after transplant can lower the risk of your disease coming back.
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 Year 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have undergone T cell depleted allogeneic hematopoietic stem cell transplantation at MSKCC for:
- •De novo myelodysplastic syndromes (MDS): IPSS-1 with poor risk cytogenetics or higher IPSS.
- •Acute myelogenous leukemia (AML) in first remission that required more than 1 cycle of treatment to achieve remission or with the following cytogenetic abnormalities: FLT3 mutation, deletion/monosomy of chromosome 5 or 7, MLL gene rearrangement, or more than or equal to 3 cytogenetics abnormalities. Also patients in second or greater remission.
- •Patients with Secondary MDS/AML.
- •Patients will be considered eligible for the study if after transplant they achieved hematologic (<5% blasts) and cytogenetic remission.
- •Patients will be eligible to enter the study between 60-120 days post transplant.
- •Age: pediatrics and adults patients - 1 year old-75 years old.
- •Karnofsky performance status >=60% for patients >16yo and Lansky performance status >=60% for patients ≤16yo
- •Stable blood counts (ANC>1000/uL, Hb>8gr/dL, Plt>50,000/ uL) not supported by transfusions.
- •Renal: Serum creatinine <1.5 ULN
- •Hepatic: <3xULN ALT and <1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
- •Cardiac: Adequate cardiac function measured by LVEF>50%. If asymptomatic, pretransplant echocardiogram is adequate. If symptomatic, echocardiogram needs to be repeated.
- •Each patient must be willing to participate as a research subject and must sign an informed consent form.
排除标准
- •Patients will be excluded from the trial if at time of enrollment:
- •Active uncontrolled bacterial, fungal or viral infection.
- •Evidence of uncontrolled graft-versus-host disease.
- •Pulmonary: new onset hypoxia
- •Known or suspected hypersensitivity to 5'-azacitadine or mannitol.
- •Evidence of residual disease either by increased blasts count (>5%) or persistence of previous known cytogenetics abnormalities.
- •Peripheral blood neutrophil chimerism: less than 95% donor.
研究组 & 干预措施
low dose 5'-azacitidine
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
干预措施: low dose 5'-azacitidine (Drug)
结局指标
主要结局
Relapse Rate
时间窗: 2 years
Relapse of MDS or AML will be analyzed as to type and genetic origin of the leukemic cells. These will be defined by morphologic and/or cytogenetic criteria: an increasing number of blasts in the marrow over 5%, by presence of circulating blasts, or by presence of blasts in any extramedullary site as well as presence of previous cytogenetic abnormalities. Other studies assessing for MRD, FACS and FISH assays will be evaluated but would not be considered disease relapse if positive since they are experimental.
次要结局
- Overall Survival(2 years)
- Number of Participants Evaluated for Treatment Safety(2 years)