A Multi-national Open-label Phase II Study of the JAK Inhibitor INC424 in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24
研究概览
简要总结
The objective of this study was to determine the efficacy of INC424 as assessed by reduction in spleen volume in patients with primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF. The safety and tolerability of INC424 and the effects of INC424 on patient reported outcomes and the duration of response as assessed by reduction in spleen volume was also assessed.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years or older
- •Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF
- •Enlarged spleen, measuring 5 cm or greater from the costal margin
- •Must have two or more of the following risk factors:
- •Over 65 years old
- •Have the following symptoms often associated with MF: loss of weight, fever, night sweats
- •Have a low red blood cell count (anemia - hemoglobin < 10 g/dL)
- •Have a high white blood cell count (history of white blood cell count > 25,000/uL)
- •Have high circulating blasts (> or = 1%) as measured by blood tests
- •Should have circulating blasts <10% (as measured by blood tests)
- •Should be capable of self-care
- •Should have adequate bone marrow reserve
- •Should not have the option of stem cell transplantation
- •Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering the study
- •Had no prior treatment with another JAK inhibitor
排除标准
- •Does not have adequate liver or kidney function (as measured by blood tests)
- •Has an active infection (bacterial, viral, etc.)
- •Has active hepatitis A, B, or C or positive for HIV
- •Has another cancer that needs active intervention
- •Had a history of bleeding disorder
- •Had a history of very low platelet counts (as measured by blood tests) not related to treatment of MF
- •Had radiation of the spleen within 1 year of joining the study
- •Does not have adequate heart function
- •Sufficient time has elapsed between stopping previous treatment for MF and joining the study
- •Females who are pregnant or breast-feeding
- •Not able to sign informed consent
- •Has any other active medical conditions that the doctor deems may compromise your safety or ability to join in the study
研究组 & 干预措施
Ruxolitinib
Ruxolitinib was taken twice daily, unless instructed. Starting dose 15 mg BID for patients with baseline platelet count of 100,000/μL to 200,000/μL (inclusive) or 20 mg BID for those with baseline platelet count >200,000/μL (approximately 12 hours apart: morning and night), increased or decreased per standardized dosing paradigm.
干预措施: Ruxolitinib (Drug)
结局指标
主要结局
Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24
时间窗: 24 weeks
The primary measure of spleen size was by MRI. MRIs were performed with a body coil because the objective was to measure organ volume only, not to assess for lesions. MRIs were performed by local radiologists who were instructed not to provide a quantitative measure of spleen volume, but could provide a qualitative assessment such as enlarged, smaller, larger, etc. The scans from an individual patient were to be read by a central reader upon transfer from the site radiologist.
次要结局
- Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Each Scheduled Time Point - Best Response(Weeks 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, at any time point)
- Change in EORTC QLQ-C30 Scores From Baseline in at Week 24(Baseline, Week 24)
- Kaplan Meier Estimates of Duration of Response of at Least ≥ 35% Reduction From Baseline in Spleen Volume Per Kaplan Meier Estimates(Weeks 12, 24, 36, 48, 60, 72, 84, 96, 120, 144, 168, 192, 216, 240)
- Change in Total Symptom Score From Baseline at Week 24 as Measured by Seven-day Modified MFSAF v2.0(Baseline, Week 24)