Children's Oncology Group Pediatric Early Phase Clinical Trial Network

儿童肿瘤学组儿科早期临床试验网络

基本信息

批准号：
9789854
负责人：
Brenda J. Weigel
金额：
$ 463.96万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
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/9789854
关键词：
Adolescent Adult Adverse event Biological Biological Models Biology Characteristics Child Childhood Clinical Pharmacology Clinical Research Clinical Trials Clinical Trials Network Cohort Studies Common Terminology Criteria for Adverse Events Complex Country Cytotoxic agent DNA Sequence Alteration Data Databases Development Dose Drug Kinetics Drug effect disorder Enrollment Ensure Evaluation Foundations Genes Genomics Image Informatics Infrastructure Institution International Laboratory Research Laboratory Study Lead Malignant Childhood Neoplasm Malignant Neoplasms Mission Modeling Molecular Profiling Molecular Target New Agents Outcome Parents Pathway interactions Patient Outcomes Assessments Patients Pediatric Neoplasm Pediatric Oncology Pediatric Oncology Group Peer Review Pharmacogenetics Pharmacology Pharmacology Study Phase Pilot Projects Process Proxy Reporting Resources Signal Transduction Site Symptoms System Therapeutic Toxic effect anti-cancer therapeutic antitumor effect base cancer therapy cohort drug disposition early phase trial exome sequencing imaging modality imaging study improved novel novel anticancer drug patient population phase 3 testing pre-clinical response targeted agent targeted treatment transcriptome sequencing treatment strategy tumor tumor DNA

项目摘要

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.

儿科早期临床试验网络 (PEP-CTN) 的使命是识别和通过合理和有效的方法，开发治疗儿童和青少年癌症的有效新药高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、药理学和影像学研究，以进一步了解引入治疗癌症儿童的新药物的处置和作用。每年，PEP-CTN 都会招收大约 120 名患有癌症的儿童和青少年。在 21 个核心主要儿科肿瘤中心进行新型抗癌药物的临床试验国家。网络机构通过同行评审过程选出，并作为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护自己的行政和运营基础设施，以确保快速专门且复杂的早期临床试验的开发、实施和报告。 PEP-CTN 拥有进行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学系统促进反应和相关影像研究的转移，以供中央审查和分析。 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) 至通过使用2期扩展队列快速获得初步疗效信号试点研究，以便为将在 COG 站点进行的肿瘤特异性试验提供信息； 3) 至进行基因组分析，包括单基因研究或基因组，以确定合适的接受靶向药物早期研究的患者； 4) 识别肿瘤的关联使用基因组分析（例如全外显子组）对新药物的反应特征测序和RNA测序； 5) 结合药理学和生物学终点，包括循环肿瘤 DNA、其他转化实验室研究和成像方式，进入早期研究，以增强我们对新药及其效果的了解儿童癌症靶向治疗的开发是当务之急，因为它提供了更有效、毒性更小的治疗方法的前景。