Pilot Study of Everolimus in the Treatment of Neoplasms in Patients With Peutz-Jeghers Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- To determine the response rate of Everolimus in patients with advanced cancer and PJS.
研究概览
简要总结
In this pilot study the investigators will treat all patients known with Peutz-Jeghers syndrome (PJS) who are diagnosed with advanced malignancies with everolimus 10mg daily until disease progression. Most patients with PJS have an inherited LKB1 mutation leading to aberrant m-TOR activity. Their risk to develop malignancies or intestinal polyps is probably related to this constitutive mTOR signaling. The hypothesis is that mTOR inhibition is an effective anticancer treatment in PJS patients with advanced malignancies.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
afinitor
10mg afinitor daily orally
干预措施: Everolimus (Drug)
结局指标
主要结局
To determine the response rate of Everolimus in patients with advanced cancer and PJS.
时间窗: During treatment, expected avarage of 12 months
Determined with regular radiological scans once every 9 weeks and measured following RECIST 1.1
次要结局
- To determine the overall survival of PJS patients treated with everolimus for advanced malignancies(avarage of 18 months)
- To determine the time to progression of PJS patients treated with everolimus for advanced malignancies.(During treatment, expected avarage of 12 months)
- To determine if there is an association between measured drug blood levels and treatment outcome measured as response to treatment determined by RECIST(During treatment, expected avarage of 12 months)
- To assess markers for activated mTOR pathway (including phospho-S6 and phospho-4E BP1) in all pre-treatment tissue specimens and collected specimens during treatment and correlate with response to treatment.(During treatment, expected avarage of 12 months)
- To determine the safety and toxicity of Everolimus in this patient population(During treatment, expected avarage of 12 months)
研究者
Heinz-Josef Klumpen
MD
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)