A Phase II Study of 5-Azacitidine (5AC) in Combination With Sargramostim (GM-CSF) as Maintenance Treatment, After Definitive Therapy With Either Stem Cell Transplant (SCT) or Cytarabine-based Chemotherapy, in Patients With Poor-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Two-year Relapse Free Survival of Patients
研究概览
简要总结
To determine the impact of maintenance therapy in patients with MDS/AML in remission.
详细描述
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy.
In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed by two years of follow-up.
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
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age > 6 months
- •Initial diagnosis of poor -risk AML or MDS (defined in section 3.2), treated with either stem cell transplant or cytarabine-based consolidation chemotherapy, within the past 60-185 days
- •ECOG performance status 0-2
- •No morphologic evidence of leukemia or active MDS as determined by JHH Hematopathologist independent review of a bone marrow aspirate and biopsy done following the completion of therapy and within 14 days prior to enrollment
- •Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
- •No evidence of extramedullary leukemia, such as CNS or soft tissue involvement
- •Adequate end organ function as measured by the following: AST and ALT < 4 x normal, total serum bilirubin < 2 x upper limit normal (unless due to hemolysis, Gilbert's syndrome, or ineffective erythropoiesis), creatinine < 2 x upper limit of normal
- •Ability to give informed consent
- •In agreement to use an effective barrier method of birth control to avoid pregnancy during the study and for a minimum of 30 days after study treatment, for all male and female patients who are fertile
排除标准
- •Patients with untreated or uncontrolled infections
- •Patients with untreated or uncontrolled grade 3 or 4 GVHD
- •Pregnancy and lactation
- •Concurrent use of any other investigational agents.
- •Known HIV-positive patients.
- •Known hypersensitivity to 5AC or GM-CSF
研究组 & 干预措施
Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
干预措施: Azacitidine (Drug)
Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
干预措施: Sargramostim (Biological)
Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
干预措施: Azacitidine (Drug)
Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
干预措施: Sargramostim (Biological)
Standard consolidation
Azacitidine and sargramostim after standard consolidation
干预措施: Azacitidine (Drug)
Standard consolidation
Azacitidine and sargramostim after standard consolidation
干预措施: Sargramostim (Biological)
结局指标
主要结局
Two-year Relapse Free Survival of Patients
时间窗: 2 year
To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
次要结局
- Hematologic Toxicity as Determined by Anemia(1 year)
- One-year RFS(1 year)
- Overall Survival(2 years)