ADVL0319 PHASE I STUDY OF CC-5013 (LENALIDOMIDE) IN PEDIATRIC PATIENTS

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CC-5013的新药或来那度胺在实体瘤患者和骨髓增生异常综合征患者中的安全性。CC-5013通过阻断某些细胞过程来阻止癌细胞生长，从而导致细胞死亡。目前还没有CC-5013在儿童身上的试验。这项研究的目的是测试CC-5013的安全性，看看它对儿童和儿童癌症有什么(好的和坏的)影响，并找到CC-5013可以给实体瘤儿童而不会引起严重副作用的最高剂量，了解人体如何处理药物，更好地了解肿瘤细胞中与健康细胞不同的功能，并研究CC-5013在实体瘤和MDS患者中的抗肿瘤作用。儿童每天服用CC-5013，为期21天，在下一个疗程开始前有7天的休息期(每个疗程28天)。参与这项研究的患者可以服用CC-5013，只要他们的癌症没有恶化，只要没有不良副作用发生，最多12个疗程。参加这项研究的儿童将在治疗期间接受标准的体检，如体检、血液测试、扫描或其他测试，以评估他们的肿瘤。患者将被给予选择采取血液样本进行药代动力学和测试，以了解CC-5013对免疫功能的影响。

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 new drug called CC-5013, or lenalidomide in solid tumor patients and in patients with myelodysplastic syndrome. CC-5013 works to stop cancer cells from growing by blocking certain cell processes, causing the cell to die. There have been no trials of CC-5013 in children yet. The goals of this study are to test the safety of CC-5013, to see what effects (good and bad) it has on children and children's cancer, and to find the highest dose of CC-5013 that can be given to children with solid tumors without causing severe side effects, to learn how the body handles the drug, to better understand the functions in tumor cells that differ from healthy cells and to study the anti tumor effect of CC-5013 in solid tumor and MDS patients. CC-5013 will be given to children every day for 21 days, with a period of 7 days to rest before the next course begins (each course is 28 days). Patients on this study can be given CC-5013 as long as their cancer is not getting worse, and as long as no bad side effects happen, for up to 12 courses. Children on this study will be given standard medical exams during therapy, such as physical exams, blood tests, and scans or other tests to evaluate their tumor. Patients will be the given the option to have blood samples taken for pharmacokinetics and for tests to learn what CC-5013 does to immune functions.