Children's Oncology Group Pediatric Early Phase Clinical Trial Network

儿科早期临床试验网络（PEP-CTN）的使命是识别和 开发有效的新药物，为儿童和青少年癌症，通过合理和 有效的临床和实验室研究。PEP-CTN临床试验将纳入相关的 基因组学，生物学，药理学和成像研究，以进一步了解 新药物引入癌症儿童治疗的处置和作用。 每年，PEP-CTN将招募大约120名患有癌症的儿童和青少年， 在21个核心首要儿科肿瘤学研究中心进行的新型抗癌药物临床试验 全国这些网络机构是通过同行审查程序选定的， 儿科肿瘤学新药开发的国家和国际模式。PEP- CTN将利用母公司儿童肿瘤学的数据库基础设施和资源 集团，同时维持其自身的行政和运营基础设施，以确保快速 开发、实施和报告专业和复杂的早期临床试验。 PEP-CTN拥有开展转化生物学的专业知识和资源， 药代动力学和药物遗传学研究，并利用最先进的信息系统 便于转移缓解和相关影像学检查，以供中心审查， 分析。 PEP-CTN的主要具体目标是：1）安全有效地引入新的抗癌药物 通过开展早期临床试验，将药物引入儿科环境; 2） 通过使用II期扩展队列迅速获得初步疗效信号， 试点研究，以便为将在COG研究中心开展的肿瘤特异性试验提供信息; 3） 进行基因组分析，包括单基因研究或基因面板，以确定适当的 患者进行靶向药物的早期研究; 4）确定肿瘤 使用全外显子组等基因组分析， 测序和RNA测序; 5）为了结合药理学和生物学终点， 包括循环肿瘤DNA、其他转化实验室研究和成像方式， 以加强我们对新药物及其效果的了解 关于肿瘤儿童癌症的靶向治疗的发展是一个高度优先事项，因为它提供了 更有效和毒性更小的治疗方法的前景。

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 premier pediatric oncology sites throughout the country. 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 will leverage 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.