PBTC-018: A PHASE I TRIAL OF CC-5013 (LENALIDOMIDE)

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 这是一项在复发性或难治性脑肿瘤儿童中进行的CC-5013儿科I期试验，旨在确定CC-5013的MTD和安全性特征。 CC-5013将每天口服一次，持续21天，为期28天。 它与沙利度胺相似，具有广泛的药理学、抗血管生成和免疫学作用。 如果受试者没有出现不可接受的毒性或疾病进展，课程可以持续两年。 本研究的目的是： 估计复发性或难治性原发性CNS肿瘤儿童口服CC-5013的MTD，每日一次，持续21天，疗程为28天 描述CC-5013在复发性或难治性原发性CNS肿瘤儿童中的毒性特征并确定剂量限制性毒性 描述CC-5013在儿童和青少年中的药代动力学特征 评价CC-5013治疗患者中循环内皮细胞（CEC）和循环内皮细胞前体（CEP）的变化，并研究CEC和CEP变化与血管生成相关蛋白质（包括血小板反应蛋白、b-FGF、TNF-α、IL-12、IL-8和VEGF）的血浆、血清和尿液水平之间的相关性，并将这些变化与MR灌注和临床结果的变化相关联 评价治疗期间MR波谱、MR灌注和弥散的变化。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a pediatric phase I trial of CC-5013 performed in children with recurrent or refractory brain tumors, to determine the MTD and safety profile of CC-5013. CC-5013 will be given orally once a day for 21 days of a 28-day course. It is similar to thalidomide and has a wide variety of pharmacologic, antiangiogenic and immunologic effects. Courses may continue for two years provided the subject does not develop unacceptable toxicity or disease progression. The objectives of this study are: To estimate the MTD of oral CC-5013 administered to children with recurrent or refractory primary CNS tumors once daily for 21 days of a 28 day course To describe the toxicity profile and define the dose-limiting toxicity of CC-5013 in children with recurrent or refractory primary CNS tumors To characterize the pharmacokinetics of CC-5013 in children and adolescents To evaluate changes in circulating endothelial cells (CECs) and circulating endothelial cell precursors (CEPs) in patients treated with CC-5013, and to investigate the correlation between changes in CECs and CEPs, plasma, serum and urine levels of proteins associated with angiogenesis including thrombospondin, b-FGF, TNF- , IL-12, IL-8 and VEGF, and correlate these changes with changes in MR perfusion and clinical outcome To evaluate changes in MR spectroscopy, MR perfusion and diffusion during treatment.