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

该子项目是利用 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来源获得主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 这项I期研究测试了一种名为CC-5013或来那度胺的新药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，导致细胞死亡。 目前还没有在儿童中进行CC-5013的试验。 这项研究的目的是测试CC-5013的安全性，看看它对人体有什么影响。（好的和坏的）它对儿童和儿童癌症的影响，并找到最高剂量的CC-5013，可以给患有实体瘤的儿童，而不会引起严重的副作用，了解身体如何处理药物，为了更好地了解肿瘤细胞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。 CC-5013将每天给孩子服用21天，在下一个课程开始前有7天的休息时间（每个课程为28天）。 在这项研究中，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。这项研究中的儿童将在治疗期间接受标准的医学检查，如体格检查，血液检查和扫描或其他检查，以评估他们的肿瘤。 患者可以选择采集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.