NCT01158300已完成1 期
Phase I and Pharmacokinetic Trial of PTC299 in Pediatric Patients With Refractory or Recurrent CNS Tumors
Pediatric Brain Tumor Consortium16 个研究点 分布在 1 个国家目标入组 28 人开始时间: 2010年11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
- 28
- 试验地点
- 16
- 主要终点
- Adverse events
研究概览
简要总结
RATIONALE: PTC299 may stop the growth of tumor cells by blocking blood flow to the tumor.
PURPOSE: This phase I trial is studying the side effects and the best dose of PTC299 in treating young patients with recurrent or refractory primary central nervous system tumors.
详细描述
OBJECTIVES:
Primary
- To estimate the maximum-tolerated dose and the recommended phase II dose of VEGF inhibitor PTC299 (PTC299) in pediatric patients with recurrent or progressive primary central nervous system (CNS) tumors.
- To evaluate and characterize the adverse events associated with this regimen in these patients.
- To evaluate and characterize the pharmacokinetics and pharmacodynamics of this regimen in these patients.
Secondary
- To investigate the relationships between PTC299 plasma exposure and other outcomes measures.
- To evaluate the antitumor activity of this regimen in these patients.
- To evaluate changes in angiogenic and inflammatory markers in the blood and the relationship between these changes and other outcome measures.
- To obtain preliminary evidence of biologic activity of PTC299 by using magnetic resonance diffusion to assess tumor cellularity.
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
- 3 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histologically confirmed diagnosis of primary central nervous system (CNS) malignancy at time of diagnosis or recurrence
- •Histology verification not required for intrinsic brain stem tumors and optic pathway gliomas
- •Must have radiographic evidence of progression
- •Recurrent, progressive, or refractory disease to standard therapy and for which there is no known curative therapy
- •PATIENT CHARACTERISTICS:
- •Karnofsky performance status (PS) 50-100% (patients > 16 years of age) OR Lansky PS 50-100% (patients ≤ 16 years of age)
- •Body weight ≥ 15 kg and ≤ 100 kg
- •Patients with neurological deficits allowed provided they are stable for ≥ 1 week
- •Able to swallow capsules
- •ANC ≥ 1,000/μL (unsupported)
- •Platelet count ≥ 100,000/μL (unsupported)
- •Hemoglobin ≥ 8 g/dL (may be supported)
- •Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m^2 OR serum creatinine normal based on age as follows:
- •0.8 mg/dL (≤ 5 years of age)
- •1.0 mg/dL (> 5 to ≤ 10 years of age)
- •1.2 mg/dL (> 10 to ≤ 15 years of age)
- •1.5 mg/dL (> 15 years of age)
- •Urine protein/creatinine ratio < 1.0
- •Total bilirubin ≤ 1.5 times upper limit of normal (ULN)
- •ALT and AST ≤ 2.5 times ULN
- •Albumin ≥ 2.5 g/dL
- •PT and activated PTT ≤ 1.2 times ULN
- •Not pregnant or nursing
- •Negative pregnancy test
- •Fertile patients must use effective contraception
- •No clinically significant unrelated systemic illness that would compromise the patient's ability to tolerate protocol therapy, or would likely interfere with the study procedures or results, including any of the following:
- •Serious infections including ongoing systemic bacterial, fungal, or viral infection
- •Significant cardiac, pulmonary, hepatic, or other organ dysfunction
- •Willing and able to comply with schedule visits, drug administration plan, laboratory tests, including pharmacokinetic and pharmacodynamic assessments, or other study procedures
- •No known coagulopathy or bleeding diathesis
- •No known history of drug-induced liver injury
- •No CNS, pulmonary, gastrointestinal, or urinary bleeding within the past month
- •No uncontrolled systemic hypertension (systolic BP or diastolic BP > 95% percentile for age)
- •No alcohol or drug addiction
- •Able to tolerate periodic MRI scans and gadolinium contrast
- •PRIOR CONCURRENT THERAPY:
- •See Disease Characteristics
- •Recovered from the acute toxic of all prior therapy (excluding alopecia and neurotoxicity)
- •At least 3 weeks since prior myelosuppressive anticancer chemotherapy (6 weeks for nitrosourea)
- •At least 14 days since prior investigational or biological agent
- •At least 3 half-lives since prior biological agents that have a prolonged half-life
- •At least 3 half-lives since prior monoclonal antibody
- •At least 2 weeks since prior local palliative radiotherapy
- •At least 6 weeks since prior total-body irradiation, craniospinal radiotherapy, or radiotherapy to ≥ 50% of the pelvis
- •At least 90 days since prior allogeneic bone marrow transplantation
- •No active graft-versus-host disease
- •Concurrent dexamethasone or other corticosteroids allowed provided dose is stable for ≥ 7 days
- •At least 1 week since prior colony-forming growth factors (e.g., filgrastim, sargramostim, erythropoietin)
- •At least 14 days since long-acting colony-forming growth factor formulations (e.g., pegfilgrastim)
- 另有 4 项未显示
排除标准
- 未提供
结局指标
主要结局
Adverse events
时间窗: From the first day of treatment until 30 days after the last dose
Maximum-tolerated dose
时间窗: First four weeks of treatment
次要结局
- Percentage of study participants with complete response or partial response to the study treatment(Every 8 weeks)
- Pharmacokinetics(Day 1 and day 28 of course 1)
- Change from baseline in blood angiogenic markers and cytokines at discontinuation or completion of treatment(Before the first dose of drug on day 1 of course 1 and at the discontinuation or completion of treatment)
研究者
研究点 (16)
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