PEP-CTN EHR to RAVE supplement

PEP-CTN项目摘要/摘要： 儿科早期临床试验网络(PEP-CTN)的任务是确定和 为患有癌症的儿童和青少年开发有效的新药物，通过合理和 高效的临床和实验室研究。PEP-CTN临床试验将纳入相关 基因组学、生物学、药理学和成像研究，以加深对 儿童癌症治疗中引入的新药物的处置和作用。 每年，PEP-CTN将招收大约120名癌症儿童和青少年参加 新型抗癌药物在21个核心成员和21个非核心成员的临床试验 遍布美国、加拿大和澳大利亚的网站。网络机构是通过一个 同行评审程序，并作为国家和国际新代理商开发的典范 儿科肿瘤学。PEP-CTN利用数据库基础架构和 亲子肿瘤学小组维持自己的管理和运作 基础设施，以确保快速开发、实施和报告专门和 复杂的早期临床试验。PEP-CTN拥有专门知识和资源来开展 翻译生物学、药代动力学和药物遗传学研究，并利用最先进的 信息学系统，以促进响应和相关成像研究的传输 集中审查和分析。 PEP-CTN的主要具体目标是：1)安全有效地引入新型抗癌药物 通过进行早期临床试验将药物引入儿科环境；2) 通过使用二期扩展队列快速获得初步疗效信号 先导性研究，以告知将在COG地点进行的肿瘤特异性试验；3) 进行基因组分析，包括单基因研究或基因小组，以确定合适的 患者进行靶向药物的早期研究；4)确定肿瘤的相关性 利用全外显子组等基因组分析方法对新制剂的反应特性 测序和RNA测序；5)结合药理学和生物学终点， 包括循环肿瘤DNA，其他翻译实验室研究，以及成像方式， 进入早期研究阶段，以加强我们对新制剂及其效果的了解 在肿瘤上..开发针对儿童癌症的靶向治疗是一个高度优先的问题，因为它提供了 更有效、毒性更低的治疗方法的前景。

PEP-CTN Project Summary/Abstract: The mission of the Pediatric Early Phase Clinical Trials Network (PEP-CTN) is to identify and develop effective new agents for children and adolescents with cancer, through rational and efficient clinical and laboratory research. PEP-CTN clinical trials will incorporate correlative genomics, biology, pharmacology, and imaging studies to further the understanding of the disposition and action of new agents introduced into the treatment of children with cancer. Annually, the PEP-CTN will enroll approximately 120 children and adolescents with cancer onto clinical trials of novel anticancer agents at the 21 core-member sites and 21 non-core member sites throughout the US, Canada, and Australia. The network institutions are selected through a peer review process, and serve as a national and international model for new agent development in pediatric oncology. The PEP-CTN leverages the database infrastructure and resources of the parent Children’s Oncology Group while maintaining its own administrative and operational infrastructure to ensure rapid development, implementation, and reporting of specialized and complex early phase clinical trials. The PEP-CTN has expertise and resources for the conduct of translational biology, pharmacokinetic, and pharmacogenetic studies, and utilizes state-of-the-art informatics systems to facilitate the transfer of response and correlative imaging studies for central review and analyses. The PEP-CTN's primary specific aims are: 1) To safely and efficiently introduce novel anticancer agents into the pediatric setting through the conduct of early phase clinical trials; 2) To expeditiously obtain preliminary efficacy signals through use of phase 2 expansion cohorts and pilot studies in order to inform tumor specific trials that will be conducted across COG sites; 3) To perform genomic analyses, including single gene studies or gene panels, to identify appropriate patients for early phase studies of targeted agents; 4) To identify associations of tumor characteristics with response to new agents using genomic analyses such as whole exome sequencing and RNA sequencing; 5) To incorporate pharmacologic and biologic endpoints, including circulating tumor DNA, other translational laboratory studies, and imaging modalities, into early phase studies, in order to enhance our understanding of the new agents and their effect on tumors.. Development of targeted therapy for childhood cancer is a high priority as it offers the prospect of more efficacious and less toxic therapeutics.