Therapy of Hypereosinophilic Syndrome, Polycythemia Vera, Atypical CML or CMML With Platelet Derived Growth Factor (PDGF-R) Fusion Genes, or Mastocytosis With Imatinib Mesylate (STI571)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 125
- 试验地点
- 1
- 主要终点
- Number of Participants With a Complete Response (CR)
研究概览
简要总结
The goal of this clinical research study is to see if Gleevec, known as imatinib mesylate (STI571), can improve the disease condition in patients with hypereosinophilic syndrome, polycythemia vera, atypical CML or CMML with PDGF-R fusion genes, or mastocytosis.
详细描述
Imatinib mesylate is a chemical compound that blocks a protein that is responsible for a certain form of leukemia. However, imatinib mesylate also blocks other important proteins that may be responsible for other blood diseases such as myeloproliferative disorders.
Patients in this study will take 4 tablets of imatinib mesylate by mouth every day. Patients with HES will take 1 tablet daily to begin, and may go up to 4 tablets daily depending on response. Imatinib mesylate should be taken each morning at breakfast with a large glass of water. Bottles containing the tablets will be given to the patient every month. Unused supplies must be returned at the end of the study. Patients taking oral hydroxyurea to control their blood counts, can continue it during the first month of imatinib mesylate treatment, but must stop taking it from then on.
After completing 2 months of therapy, response to imatinib mesylate will be evaluated. If the response is good, treatment with imatinib mesylate alone will be continued. If the response is not good, the dose of imatinib mesylate will be increased to 8 tablets daily (4 in the morning and 4 in the evening) or may be decreased to 3 tablets daily. This will be based on how the drug is tolerated. Treatment may be continued for up to one year, or as long as it is judged best to control the leukemia.
Patients will be asked to visit their doctor for a physical exam and vital signs. The frequency of doctor visits will vary depending on physical condition.
Blood tests (about 2 teaspoons) will be done once each year. The blood samples will be used for routine lab tests. A bone marrow sample will also be taken to check and measure cells related to the disease after 3 - 4 months, then every 3-6 months in the first year. If the initial bone marrow sample does not show disease, repeated bone marrows will not be done.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have 1 of the following hematopoietic malignancies: Hypereosinophilic syndrome (HES), Polycythemia vera (PV), Atypical CML or CMML with PDGF-R fusion genes, Mastocytosis, Serum bilirubin less than 2 mg%, serum creatinine less than 2 mg% unless abnormality is considered due to hematologic malignancy by investigator, Eastern Cooperative Oncology Group (ECOG) performance status < 3, life expectancy > 12 wks,
- •continued from above. Participants must sign informed consent indicating they are aware of the investigational nature of the study, in keeping with policies of the hospital, women of pregnancy potential must practice birth control. Women and men must continue birth control for the duration of the trial and at least 3 months after the last dose of study drug. Inclusion of women and minorities: As per NIH policy, women and members of minorities will be included as they are referred in the relevant populations.
- •continued from above. There are no exclusions of women or minorities based on the study objectives, New York Heart Association (NYHA) Class <3.
排除标准
- 未提供
研究组 & 干预措施
Imatinib
Imatinib mesylate 400 mg orally daily, and in HES patients start with imatinib mesylate 100 mg orally daily.
干预措施: Imatinib Mesylate (Gleevec) (Drug)
结局指标
主要结局
Number of Participants With a Complete Response (CR)
时间窗: after 2 months of therapy, up to 1 year.
Acute myeloid leukemia (AML), Myelodysplastic Syndromes (MDS): CR=Normalization peripheral blood \& bone marrow with 5% or less blasts; normo- or hypercellular marrow; Absolute Neutrophil Count (ANC) \> 1.0 x 10\^9/L, \& platelet count \>100 x 10\^9/L; or CR marrow=As per CR but platelet count \< 100 x 10\^9/L. Agnogenic myeloid metaplasia (AMM) \& CMML: CR=Absence of signs/symptoms of disease; White blood count between 1 to 10 x 10\^9/L with no peripheral blasts, promyelocytes, or myelocytes and normalization of bone marrow (\< 5% blasts in normocellular or hypercellular marrow) for 4+ weeks. PV: CR=normalization of hemoglobin/hematocrit without need for phlebotomies, disappearance all signs/symptoms of disease. HES: CR=disappearance of eosinophilia (\</=10%), disappearance signs/symptoms of disease. Mastocytosis: CR=disappearance of mast cell infiltrates in affected organs, decrease of serum tyrptase levels to \<20 ng/ml, \& disappearance of SM-associated organomegaly.
次要结局
- Overall Survival(From the start of therapy to death or disease progression, assessed up to 12 years and 5 months)
- Duration of Response(From response evaluation (first evaluation following 2 months therapy) to disease progression or death or until disease progression whichever occurs first, up to 12 years and 5 months)