Phase I Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 38
- 试验地点
- 1
- 主要终点
- Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer
研究概览
简要总结
The purpose of this study is to test whether perifosine- a drug that inhibits the protein AKT, and has had some success in the treatment of adult cancers- is safe and effective in treating cancer. The investigators want to find out what effects, good and/or bad, it has on the patient and the cancer. The investigators are testing different dose schedules of perifosine and the patient will be asked to partake in one of the dose schedules.
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
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Any solid tumor that failed standard therapy.
- •Patient must have evidence for tumor by CT, MRI, PET scan, MIBG scan, serum markers, or tissue sampling.
- •Age ≤ 21 years.
- •Karnofsky/Lansky performance status ≥ 50% (Karnofsky score for age> 16 years and Lansky score for age ≤ 16 years)
- •ANC≥ 1000 at least 24 hours off GCSF
- •Platelets ≥ 75k at least one week off platelet transfusions
- •Hg≥ 8g/dL at least one week off PRBC transfusion
- •AST ≤ 3 x the upper limit of normal
- •ALT ≤ 3 x the upper limit of normal
- •Total bilirubin ≤ 2.0 mg/dl
- •serum creatinine ≤ 1.5 x the upper limit of normal for age, or calculated creatinine clearance or nuclear GFR ≥ 70 ml/min/1.73 m
- •≥ 3 weeks since last non-nitrosourea chemotherapy
- •≥ 6 weeks since last nitrosoureas
- •≥ 4 weeks since last RT
- •Patients must agree to practice adequate contraception. Females of childbearing potential must have a negative B-HCG pregnancy test documented within 14 days prior to drug initiation. Females must not be breast feeding.
- •Patients must be able to swallow tablets whole
排除标准
- •Pregnancy
- •Patients must not have active infection or serious intercurrent medical illness.
- •HIV-Positive patients receiving combination anti-retroviral therapy are excluded from the study due to possible retro-viral drug interactions. HIV testing not required.
- •Patients must not be taking EIAEDs. If patients were previously EIAEDs that have been discontinued, patients must have been off the agent for at least 2 weeks prior to registration.
研究组 & 干预措施
perifosine
This will be a dose escalation study to determine the maximum tolerated dose (MTD) of perifosine alone in recurrent/progressive pediatric tumors. A standard 3+3 dose escalation design will be employed with 3-6 patients at each dose level.
干预措施: perifosine (Drug)
结局指标
主要结局
Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer
时间窗: conclusion of the study
次要结局
- To determine whether pharmacokinetic serum levels correlate with toxicity(conclusion of the study)
- If previously resected tissue is available, determine whether molecular features predict response including Elevated PI3K/AKT/mTOR signaling, Elevated RAS/MAPK signaling, Cell cycle markers(conclusion of the study)