COG ADVL0413: A PHASE I STUDY OF THE RAF KINASE AND RECEPTOR TYROSINE KINASE

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项I期研究测试了一种名为索拉非尼(BAY43-9006)的药物在患有难治性实体瘤或难治性白血病的儿童中的安全性。索拉非尼通过阻断蛋白质发挥作用，而蛋白质被认为是传递肿瘤生长信号的重要信号。索拉非尼最近获得了FDA的批准，用于治疗晚期肾癌，这种癌症主要发生在成年人身上。这项研究的目的是找到索拉非尼可以给实体瘤儿童使用而不会引起严重副作用的最高安全剂量，找出实体瘤儿童使用最高安全剂量索拉非尼是否对白血病儿童也安全，并了解索拉非尼会导致什么样的副作用。这项研究的其他目标包括了解身体如何处理药物，了解索拉非尼对血细胞和肿瘤细胞有什么影响，以及了解更多关于索拉非尼是否可能对儿童肿瘤有益的治疗方法。患者将每天服用两次索拉非尼。可提供长达24-28天的疗程。索拉非尼片剂必须与清澈的液体一起整个吞下。除了这些测试外，患者还可以选择在治疗的第一个疗程中采集血液样本进行药代动力学和相关研究。可选的研究还包括DEMRI。一旦发现实体瘤患者的最大剂量，一组难治性白血病儿童将接受该剂量的索拉非尼治疗。

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 phase I study tests the safety of a drug called sorafenib (BAY43-9006) in children with refractory solid tumors or refractory leukemia. Sorafenib works by blocking proteins which are thought to be important for signaling tumor growth. Sorafenib recently received FDA approval for treatment of advanced kidney cancer which occurs mostly in adults. The goals of this study are to find the highest safe dose of Sorafenib that can be given to children with solid tumors without causing severe side effects, to find out if the highest safe dose of Sorafenib in children with solid tumors is also safe for children with leukemia, and to learn what kind of side effects Sorafenib can cause. Other goals of this study include learning how the body handles the drug, learning what effects Sorafenib has on blood cells and tumor cells, and learning more about whether Sorafenib may be a beneficial treatment for pediatric tumors. The patients will be given Sorafenib twice a day. Up to 24-28 day courses of treatment may be given. Sorafenib tablets must be swallowed whole with clear liquids. In addition to these tests, patients will be the given the option to have blood samples taken during the first course of therapy for pharmacokinetics and correlative studies. Optional studies also include DEMRIs. Once the maximum dose is found in patients with solid tumors, a group of children with refractory leukemia will be given Sorafenib at this dose.