Open-label, Phase II Clinical Trial of Aplidin® (Plitidepsin) in Patients With Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia (Post-PV/ET) Myelofibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Objective Response Rate (ORR)
研究概览
简要总结
This is an open-label, Phase II Clinical Trial of Aplidin® (plitidepsin) in Patients with Primary Myelofibrosis and post polycythemia vera/essential thrombocythemia (Post-PV/ET) Myelofibrosis.
详细描述
This trial tries to assess response rate (ORR) of plitidepsin in patients with:
primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF). Besides, the study results will allow to evaluate the effect of plitidepsin on bone marrow (BM) or peripheral blood histology and to determine the quality of life (QoL) and symptoms or participant patients.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Primary Myelofibrosis (PMF) or Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis(post-ET/PV MF) as per revised World Health Organization (WHO) criteria.
- •High-risk or intermediate-2 risk Myelofibrosis (MF) as defined by the International Prognostic Scoring System (IPSS); or intermediate-I risk MF associated with symptomatic splenomegaly/hepatomegaly and/or unresponsive to available therapy.
- •At least 18 years of age, with life expectancy of ≥12 weeks.
- •Able to provide informed consent and being willing to sign an informed consent form (ICF).
- •Eastern Cooperative Oncology Group (ECOG) performance status ≤
- •Evidence of acceptable organ function within seven days of initiating study drug
排除标准
- •Previous treatment with plitidepsin.
- •Any of the following therapies within two weeks prior to initiation of study drug:
- •chemotherapy (e.g., hydroxyurea),
- •immunomodulatory drug therapy (e.g., thalidomide),
- •immunosuppressive therapy,
- •corticosteroids >10 mg/day prednisone or equivalent, or
- •erythropoietin.
- •Incomplete recovery from major surgery within four weeks of study entry.
- •Radiation therapy within four weeks of study entry.
- •Women of childbearing potential
- •Women who are pregnant or are currently breastfeeding.
- •Myopathy grade > 2
- •Known positive status for human immunodeficiency virus (HIV).
- •Active hepatitis B or C virus (HBV or HCV) infection
- •Diagnosis of another invasive malignancy
- •Any acute active infection.
- •Known hypersensitivity to the study drug or any of its formulation components (e.g., Cremophor®).
- •Treatment with any investigational product in the 30 days before inclusion in the study.
研究组 & 干预措施
Arm one
干预措施: APLIDIN (plitidepsin) (Drug)
结局指标
主要结局
Objective Response Rate (ORR)
时间窗: All patients were followed up to progressive disease, start of a new anti-cancer therapy, death or one year after the last treatment visit of the last patient, whichever occured first
Objective response rate (ORR) of plitidepsin in patients with: primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. ORR according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) response criteria (Tefferi et al., 2006) in the evaluable population: defined as a confirmed disease response, on two consecutive evaluations performed at least eight weeks apart. Overall response (OR) = Complete Response (CR) + Partial response (PR) + Clinical improvement (CI).
次要结局
- Quality of Life (QoL)(All patients were followed up to progressive disease, start of a new anti-cancer therapy, death or one year after the last treatment visit of the last patient, whichever occured first)