NCT01528384已完成1 期
A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy
Polaris Group2 个研究点 分布在 1 个国家目标入组 8 人开始时间: 2011年12月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Change in laboratory values.
研究概览
简要总结
This is a phase 1 study of pegylated arginine deiminase (ADI-PEG 20) in pediatric patients who deficient in the expression of tumor argininosuccinate synthetase, and have failed prior treatments. The study hypothesis is that this drug will be safe in pediatric patients.
详细描述
This phase 1 study will use a classic 3 dose levels by 3 patients per dose level design. Once the maximum tolerated dose is reached, additional patients may be enrolled at that dose level.
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
- 4 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prior diagnosis of failed current standard treatment(s), or AEs associated with prior systemic agent(s) that resulted in discontinuance of that agent(s).
- •Argininosuccinate synthetase deficient tumor.
- •Measurable disease as assessed by >5% blasts on bone marrow aspirate for patients with leukemia or using RECIST 1.1 criteria for solid tumors.
- •Age 4 to ≤ 18 years.
- •No prior systemic treatment for the tumor in the last 2 weeks for solid tumors and 1 week for leukemia, except hydroxyurea used only to control very high WBC.
- •Fully recovered from any prior surgery and no major surgery within 4 weeks of initiating treatment. Surgery for placement of vascular access devices is acceptable.
- •Female subjects of 10 years and older and male subjects must be asked to use appropriate contraception for both the male and female for the duration of the study.
- •No concurrent investigational studies are allowed.
- •For solid tumor subjects only: Absolute neutrophil count (ANC) >750/µL.
- •For solid tumor subjects only: Platelets >50,000/µL.
- •For solid tumor subjects only: Serum uric acid ≤ 8 mg/dL (with or without medication control).
- •Serum creatinine ≤ 1.5 x the upper limit of normal range, or, if serum creatinine >1.5 x the upper limit of normal range, then the creatinine clearance must be ≥ 60 mL/min.
排除标准
- •Serious infection requiring treatment with systemically administered antibiotics at the time of study entrance.
- •Pregnancy or lactation.
- •Expected non-compliance.
- •Uncontrolled intercurrent illness.
- •Subjects who have had any anticancer treatment prior to entering the study and have not recovered to baseline or ≤ Grade 1 AEs due to agents administered.
- •Subjects who had been treated with ADI-PEG 20 previously.
- •History of seizure disorder not related to underlying cancer.
- •Known HIV positivity.
结局指标
主要结局
Change in laboratory values.
时间窗: 2 years
Change from baseline in laboratory values, as a measure of safety.
次要结局
- Results of diagnostic imaging using RECIST criteria.(2 years)
研究者
研究点 (2)
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