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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)

To determine the impact of maintenance therapy in patients with MDS/AML in remission.

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

介入性

注册 (实际的)

25

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习地点

    • Maryland
      • Baltimore、Maryland、美国、21287
        • The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

6个月 及以上 (孩子、成人、年长者)

接受健康志愿者

有资格学习的性别

全部

描述

Inclusion Criteria:

  1. Age > 6 months
  2. 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
  3. ECOG performance status 0-2
  4. 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
  5. Peripheral blood count recovery: Neutrophil count ≥ 1000 /µL, platelet count ≥ 50x 109 /µL without platelet transfusions, and adequate hematocrit independent of red cell transfusions .
  6. No evidence of extramedullary leukemia, such as CNS or soft tissue involvement
  7. 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
  8. Ability to give informed consent
  9. 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:

  1. Patients with untreated or uncontrolled infections
  2. Patients with untreated or uncontrolled grade 3 or 4 GVHD
  3. Pregnancy and lactation
  4. Concurrent use of any other investigational agents.
  5. Known HIV-positive patients.
  6. Known hypersensitivity to 5AC or GM-CSF

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

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
实验性的:Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
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:
  • 氮杂胞苷
  • 维达扎
  • 5-氮杂胞苷
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:
  • 转基因脑脊液
实验性的:Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
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:
  • 氮杂胞苷
  • 维达扎
  • 5-氮杂胞苷
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:
  • 转基因脑脊液
实验性的:Standard consolidation
Azacitidine and sargramostim after standard consolidation
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:
  • 氮杂胞苷
  • 维达扎
  • 5-氮杂胞苷
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:
  • 转基因脑脊液

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
Two-year Relapse Free Survival of Patients
大体时间: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.
2 year

次要结果测量

结果测量
措施说明
大体时间
Hematologic Toxicity as Determined by Anemia
大体时间:1 year
Percentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
1 year
One-year RFS
大体时间:1 year
We will report the number of participants with one year RFS
1 year
Overall Survival
大体时间:2 years
Percentage of participants with overall survival at 2 years.
2 years

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

调查人员

  • 学习椅:Margaret Showel, MD、JHU

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2013年6月1日

初级完成 (实际的)

2020年6月1日

研究完成 (实际的)

2020年6月1日

研究注册日期

首次提交

2012年10月2日

首先提交符合 QC 标准的

2012年10月3日

首次发布 (估计)

2012年10月4日

研究记录更新

最后更新发布 (实际的)

2022年10月18日

上次提交的符合 QC 标准的更新

2022年9月23日

最后验证

2021年8月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

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