Phase I Study of 5-Fluorouracil in Children and Young Adults With Recurrent Ependymoma
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- Estimate the maximum tolerated dose determined using the Rolling 6 design using the CTCAEv4 to assess DLT.
研究概览
简要总结
This is a phase I study to investigate the safety and pharmacokinetics of weekly 5-fluorouracil (5-FU) administered as a bolus dose in children and young adults with recurrent or refractory ependymoma. The results from this study will inform a subsequent phase II St. Jude investigator-initiated trial.
详细描述
The initial 5-FU dosage will be 500 mg/m^2 administered on day 1 of course 1. We plan to treat a maximum of 3 cohorts of research participants (dosage levels - 0, 1, and 2) with escalating doses of 5-FU. A cycle is defined as 42 days. The first 6 weeks of therapy will constitute the dose-limiting toxicity (DLT) evaluation period.
Primary objective
- To investigate the safety and pharmacokinetics (plasma and cerebrospinal fluid) of weekly bolus dose 5-FU in children and young adults with recurrent/refractory ependymoma
- To study the safety of 500 mg/m^2 weekly bolus dose 5-FU in less-heavily pre-treated children and young adults with recurrent/refractory ependymoma.
Secondary objectives
- To document and describe toxicities associated with 5-FU administered on a weekly bolus schedule
- To document preliminary antitumor activity in participants with recurrent or refractory ependymoma treated with 5-FU
- To assess the feasibility of measuring expression level of Thymidylate Synthetase (TYMS) in formalin fixed paraffin embedded (FFPE) tumor samples using the Quantigene assay
- To evaluate the association between specific genetic polymorphisms (e.g., DPYD) and the pharmacokinetics of 5-FU
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
- 1 Month 至 21 Years（Child, 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
Treatment
Participants meeting the eligibility requirements.
Intervention: 5-fluorouracil
干预措施: 5-fluorouracil (Drug)
结局指标
主要结局
Estimate the maximum tolerated dose determined using the Rolling 6 design using the CTCAEv4 to assess DLT.
时间窗: At the end of the 6-week dose limiting toxicity observation period.
To investigate the safety and pharmacokinetics (plasma and cerebrospinal fluid) of weekly bolus dose 5-fluorouracil (5-FU) in children and young adults with recurrent/refractory ependymoma
Pharmacokinetic modeling of 5-fluorouracil concentrations
时间窗: Pharmacokinetics on day 1, day 8, and day 22 of course 1, and day 1 of course 2
To investigate the safety and pharmacokinetics (plasma and cerebrospinal fluid) of weekly bolus dose 5-fluorouracil (5-FU) in children and young adults with recurrent/refractory ependymoma
Estimate the maximum tolerated dose in less heavily pre-treated children
时间窗: At the end of the 6-week dose limiting toxicity observation period.
To investigate the safety and pharmacokinetics (plasma and cerebrospinal fluid) of weekly bolus dose 5-fluorouracil (5-FU) in children and young adults with recurrent/refractory ependymoma and in less heavily pre-treated children.
次要结局
- Descriptive report of toxicities.(Throughout treatment, up to two years per patient)
- Tumor response and progression-free survival(at the completion of therapy (2 years))
- Expression level of TYMS in FFPE tumor samples(At the end of accrual (3 years))
- Description of association between genetic polymorphism and pharmacokinetics(At the end of therapy (2 years))