MethoTRExATE in MyelOpRolifErative Neoplasms (TREATMORE) Trial
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 54
- 试验地点
- 1
- 主要终点
- MF Overall response rate
研究概览
简要总结
Low-dose MTX is a widely used, inexpensive, and safe therapy used for decades and is well tolerated by patients with rheumatologic diseases. Recently, it was identified as a type 2 JAK inhibitor. If MTX proves to be safe and tolerable with a signal of clinical activity, this could have a significant benefit to patients with MPNs. Beyond the potential benefit of adding a type 2 JAK inhibitor to current therapy, this could signal the need to study MTX in MPNs further as a monotherapy. Discovering MTX as safe and clinically effective in MPNs could be profound on both a public health and global health scale for patients who are uninsured and cannot afford more expensive novel JAK inhibitors, or for those in countries where JAK inhibitors are not available. Accordingly, the research team deems it reasonable and prudent to assess the safety and efficacy of MTX in addition to current therapy for patients with MPN. The research team will evaluate patients for spleen responses, symptom responses, and cytologic responses. Correlative data will evaluate pharmacokinetic and disease modifying activity of MTX in MPNs to inform future clinical trials.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Myelofibrosis (MF)
18 patients with MF will be enrolled
干预措施: Methotrexate (MTX) (Drug)
Polycythemia vera (PV)
18 patients with MF will be enrolled
干预措施: Methotrexate (MTX) (Drug)
Essential thrombocythemia (ET)
18 patients with MF will be enrolled
干预措施: Methotrexate (MTX) (Drug)
结局指标
主要结局
MF Overall response rate
时间窗: at 24 weeks
MF overall response rate, defined as Clinical Improvement (CI) or greater per the IWG-MRT and ELN Response Criteria for MF (2013). CI or greater is defined as changes in the spleen, hemoglobin, and symptoms.
PV and ET Overall response rate
时间窗: at 24 weeks
PV overall response rate, defined as complete response (CR) or partial response (PR) by modified 2013 ELN criteria. CR response is defined as a lasting reduction in spleen size, symptom improvement, a reduction in platelet and white cell count. As well as changes in bone marrow biopsy. PR response is defined as lasting reduction in spleen size, symptom improvement, a reduction in platelet and white cell count.
次要结局
- Adverse event Grade(Up to 48 Weeks)
- Number of Participants with Myeloproliferative Neoplasm Symptom Assessment Form Score >50%(Up to 48 Weeks)
- Anemia Response Rate(Up to 48 Weeks)
- Change in baseline hematocrit (PV cohort)(Baseline and 48 Weeks)
- Spleen Response Rate(Up to 48 Weeks)
- Change from baseline monthly phlebotomy rate (PV cohort)(Baseline and 6 months)
- Change in baseline platelet count (ET cohort)(Baseline and 48 Weeks)
- Change from baseline platelet transfusion dependence(Baseline and 48 Weeks)
- Change from baseline pRBC transfusion dependence(at 24 and 48 Weeks)
- Change from baseline in dosing of cytoreductive agents (PV and ET Cohort)(Baseline and 48 Weeks)
研究者
John Mascarenhas
Professor of Medicine
Icahn School of Medicine at Mount Sinai