Phase 1, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumors
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Determine the safety and tolerability of DCA in RMBTs.
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of oral Dichloroacetate (DCA) in the treatment of recurrent malignant brain tumors (RMBTs). RMBTs are defined as either: 1) malignant tumors, originating in the brain, that have recurred at least once or 2) malignant tumors originating elsewhere in the body that have spread to the brain at least once. Otherwise, there are no limitations to the number of prior recurrences. There are no limitations to the number or types of prior therapies.
详细描述
Malignant brain tumors are defined as any World Health Organization grade III-IV glioma and any solid tumor metastasis (spread) to the brain. Recurrent malignant brain tumors (RMBTs) are defined as either: 1) malignant tumors, originating in the brain, that have recurred at least once or 2) malignant tumors originating elsewhere in the body that have spread to the brain at least once. They share an increasing incidence, clinical and radiographic characteristics, lack of effective therapies, tendency to recur, and poor outcome. Importantly, recurrent malignant brain tumor's shared characteristics may be usefully exploited by an emerging class of biologic agents called metabolic modulators of which Dichloroacetate (DCA) is the drug in the class most thoroughly investigated clinically. DCA's mechanism of action and tolerability have been extensively demonstrated in the treatment of chronic metabolic disorders. Furthermore, the preciseness of DCA's mechanism of action appears to target abnormal tumor cell metabolism.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must be able to consent for self. Subject must have either:
- •a brain metastasis or
- •a WHO III-IV glioma that has recurred at least once. Females of child bearing age must have (-) pregnancy test.
- •Females of child bearing age must use birth control while in study.
- •Adequate organ function as determined by laboratory testing.
- •Absence of peripheral neuropathy of moderate or greater severity (physician determined).
- •Greater than 4 weeks time from previous anti-neoplastic (anti-cancer) therapy.
- •Subject must have a Karnofsky Performance Status (KPS) of greater than or equal to
- •Subject must have an ECOG performance status of less than or equal to
- •There are no limitations to the number of prior recurrences.
- •There are no limitations to the number or types of prior therapies.
排除标准
- •Medical contraindication for magnetic resonance imaging (MRI)testing.
研究组 & 干预措施
Cohort 1
Subjects are given a dose of Dichloroacetate 4mg/kg twice a day for 30 days
干预措施: Dichloroacetate (Drug)
Cohort 2
Subjects are given a dose of Dichloroacetate 12.5mg/kg twice a day for 30 days
干预措施: Dichloroacetate (Drug)
结局指标
主要结局
Determine the safety and tolerability of DCA in RMBTs.
时间窗: Within 28 days of starting DCA +/- 3 days
Oral DCA will be administered until intolerance, toxicity, radiographic progression, or death. Safety and tolerance will be assessed by reviewing available standardized clinical, radiographic, and quality of life (QOL) criteria. The safety and tolerance will also be assessed by reviewing available plasma, urine, and brain tumor tissue for metabolites of the tumor and the effects of DCA thereon.
次要结局
- Conduct an exploratory investigation of the metabolites of patients with RMBTs and the effects of DCA thereon.(One year)