Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS
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)
研究概览
详细说明
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.
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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Maryland
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Baltimore、Maryland、美国、21287
- The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:非随机化
- 介入模型:并行分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的:Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
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Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
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实验性的:Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
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Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
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实验性的:Standard consolidation
Azacitidine and sargramostim after standard consolidation
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Azacitidine will be administered days 1-5 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
Sargramostim will be administered days 1-10 of a 28 day cycle.
Treatment is planned for a total of 12 cycles.
其他名称:
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Two-year Relapse Free Survival of Patients
大体时间:2 year
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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.
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2 year
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Hematologic Toxicity as Determined by Anemia
大体时间:1 year
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Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
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1 year
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One-year RFS
大体时间:1 year
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We will report the number of participants with one year RFS
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1 year
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Overall Survival
大体时间:2 years
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Percentage of participants with overall survival at 2 years.
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2 years
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合作者和调查者
调查人员
- 学习椅:Margaret Showel, MD、JHU
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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