A Pilot Study of Trientine With Vemurafenib for the Treatment BRAF Mutated Metastatic Melanoma
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Maximum Tolerated Dose
研究概览
简要总结
The purpose of this study is to evaluate the safety of combination therapy with vemurafenib and trientine in patients with BRAF mutated metastatic melanoma.
Vemurafenib is a drug that is currently approved by the United States Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) to treat adult patients with melanoma that has spread to other parts of the body or cannot be removed by surgery. It can only be used in patients whose cancer has a change (mutation) in the "BRAF" gene.
Preclinical data suggests that use of a copper chelator (reducer) is a strategy to block cellular signaling activity which would result in anti-tumor effects (slow tumor growth). Trientine is a copper chelator and is FDA approved for the treatment of Wilson's disease (a disease of copper metabolism) and is generally well tolerated. It works by binding to copper to help remove it from the body. Trientine is not FDA approved for the treatment of melanoma and its use in this study is investigational. "Investigational" means the study drug is still being tested in research studies.
All patients will receive vemurafenib at 960mg PO twice daily with continuous dosing in combination with trientine in escalating doses. The dose of trientine will depend on what portion of the study.
In order to participate in the study, patients must test positive for the change (mutation) in the BRAF gene.
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
Vemurafenib and Trientine
干预措施: Vemurafenib and Trientine (Drug)
结局指标
主要结局
Maximum Tolerated Dose
时间窗: 1 Cycle (4 Weeks)
Maximum Tolerated Dose is defined as the highest dose level in which ≤1 of 6 patients experience a dose limiting toxicity.
次要结局
- Progression Free Survival (PFS)(2 years)
- Overall Response(1 year)
- Overall Survival (OS)(2 years)
- The change in phosphorylated Erk kinase activity relative to total Erk kinase activity(Pre-treatment; two weeks into combination therapy (day 15), and at the time of disease progression while still on combination therapy, up to 2 years)
- The change in phosphorylated MEK kinase activity relative to total MEK kinase activity(Pre-treatment; two weeks into combination therapy (day 15), and at the time of disease progression while still on combination therapy, up to 2 years)
- The change in the expression of the copper transporter CTR1(Up to 2 years)
- Incidence of hyperproliferative skin lesions, specifically cutaneous squamous cell carcinomas and keratoacanthomas(Up to 2 years)