PEP-CTN EHR to RAVE supplement

PEP-CTN EHR 至 RAVE 补充

• 批准号: 10440223
• 负责人: Brenda J. Weigel
• 金额: $ 63.85万
• 依托单位: PUBLIC HEALTH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-20 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10440223
• 关键词: Adolescent; Australia; Biological; Biology; Canada; Characteristics; Child; Childhood; Clinical Research; Clinical Trials; Clinical Trials Network; Cohort Studies; Common Terminology Criteria for Adverse Events; Complex; Data; Data Collection; Data Security; Data Set; Data Storage and Retrieval; Databases; Development; Drug Kinetics; Enrollment; Ensure; Environment; Future; Genes; Genomics; Individual; Informatics; Infrastructure; Institution; International; Laboratories; Laboratory Research; Laboratory Study; Malignant Childhood Neoplasm; Malignant Neoplasms; Maps; Medidata; Mission; Modeling; New Agents; Parents; Patients; Pediatric Oncology; Pediatric Oncology Group; Peer Review; Pharmacogenetics; Pharmacology; Pharmacology Study; Phase; Pilot Projects; Process; Protocols documentation; Reporting; Research; Resources; Role; Signal Transduction; Site; System; Testing; Therapeutic; TimeLine; Update; cancer therapy; data exchange; early phase clinical trial; exome sequencing; imaging modality; imaging study; member; novel anticancer drug; operation; pilot trial; response; skills; systems research; targeted agent; targeted treatment; tool; transcriptome sequencing; tumor; tumor DNA

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.

PEP-CTN项目摘要/摘要： 小儿早期临床试验网络（PEP-CTN）的任务是识别和 通过理性和 有效的临床和实验室研究。 PEP-CTN临床试验将纳入相关性 基因组学，生物学，药理学和成像研究，以进一步了解 引入癌症儿童治疗的新药物的性格和作用。 PEP-CTN每年将招募约120名癌症儿童和青少年 21个核心成员站点和21个非核心成员的新型抗癌药的临床试验 美国，加拿大和澳大利亚的地点。网络机构是通过 同行审查过程，并作为新代理开发的国家和国际模式 在小儿肿瘤学中。 PEP-CTN利用数据库基础架构和资源 父母的儿童肿瘤学小组，同时保持自己的行政和运营 确保专业和报告的快速发展，实施和报告的基础设施 复杂的早期临床试验。 PEP-CTN有专业知识和资源 转化生物学，药代动力学和药物遗传学研究，并利用最先进的 信息系统促进响应和相关成像研究的转移 中央审查和分析。 PEP-CTN的主要特定目的是：1）安全有效地引入新型抗癌 通过进行早期临床试验进行儿科环境的药物； 2）到 通过使用2阶段扩展队列和 试点研究是为了告知将在跨COG部位进行的特定试验； 3）到 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 对靶向药物进行早期研究的患者； 4）确定肿瘤的关联 使用基因组分析（例如整个外显子）对新药物响应的特征 测序和RNA测序； 5）要合并药物和生物终点， 包括循环肿瘤DNA，其他翻译实验室研究和成像方式， 进入早期研究，以增强我们对新药物及其影响的理解 关于肿瘤。开发针对儿童癌症的有针对性治疗是高度的重点，因为它提供了 效率更高和毒性疗法的前景。