Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Mayo Clinic
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria.
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying the side effects and how well low-dose decitabine works in treating patients with symptomatic myelofibrosis.
详细描述
OBJECTIVES:
- Determine the efficacy and safety of low-dose decitabine in patients with symptomatic primary myelofibrosis (PMF) or post essential thrombocythemic (ET) or polycythemic vera (PV) myelofibrosis.
- Analyze the ability of this drug to decrease pathologic angiogenesis and other stromal reactive features intrinsic to PMF or post ET/PV myelofibrosis.
OUTLINE: Patients receive low-dose decitabine IV over 1 hour on days 1-5. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients achieving partial remission, complete remission, or clinical improvement may receive up to 12 courses of decitabine in the absence of disease progression or unacceptable toxicity.
After completion of study therapy, patients are followed periodically for up to 3 years.
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
- 18 Years 至 120 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histological confirmation of primary myelofibrosis or post essential thrombocythemic or polycythemic vera myelofibrosis
- •Reticulin fibrosis ≥ grade 1
- •Evaluable and symptomatic disease worthy of treatment, characterized by ≥ 1 of the following:
- •Anemia, defined as hemoglobin < 11 g/dL or erythrocyte transfusion dependence
- •Palpable and symptomatic splenomegaly (palpable and symptomatic hepatomegaly is acceptable if previously splenectomized)
- •Severe, disease-related constitutional symptoms, including ≥ 1 of the following:
- •Severe night sweats
- •Weight loss
- •Bone pain
- •Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics OR prior demonstration of a lack of this translocation
- •PATIENT CHARACTERISTICS:
- •Eastern Co-operative Oncology Group (ECOG) performance status 0-3
- •Absolute neutrophil count (ANC) ≥ 1,000/mm³
- •Platelet count ≥ 50,000/mm³
- •Creatinine ≤ 2.0 mg/dL
- •Direct or total bilirubin ≤ 2.0 mg/dL
- •Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if elevation is attributed to hepatic extramedullary hematopoiesis)
- •Not pregnant or nursing
- •Negative pregnancy test
- •Fertile patients must use effective contraception
- •Not incarcerated in a municipality, county, state, or federal prison
- •No serious medical condition or psychiatric illness that would preclude signing the informed consent
- •No condition that, in the opinion of the treating physician, places the patient at unacceptable risk for study participation or confounds the ability to interpret study data
- •Able to adhere to the study visit schedule and other study requirements
- •PRIOR CONCURRENT THERAPY:
- •No other concurrent chemotherapy (e.g., hydroxyurea, thalidomide, interferon alpha, anagrelide, or other myelosuppressive agent) or experimental therapy
排除标准
- 未提供
结局指标
主要结局
Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria.
时间窗: Every 4 weeks during treatment (up to 16 weeks)
Confirmed response: objective status of CR, PR, or CI on 2 consecutive evaluations \>=4 weeks apart. CR:Complete resolution of disease-related symptoms and signs; peripheral blood count remission; normal leukocyte differential; bone marrow histologic remission. PR: All criteria for CR except the bone marrow histologic remission. CI: one of the following in the absence of both disease progression and CR/PR: minimum (MI) 20-g/L increase (INC) in hemoglobin level; MI 50% reduction in palpable splenomegaly (\>=10cm); MI 100% INC in platelet count(\>=50000x10\^9/L) or ANC (\>=0.5x10\^9/L)
次要结局
- Overall Survival(OS)(up to 3 years)
- Time to Disease Progression(up to 3 years)
- Number of Participants With Severe Adverse Events(Up to 48 weeks)
- Number of Participants With Constitutional Symptoms(Up to 48 weeks)