Pediatric Phase 1/Pilot Consortium

描述（由申请人提供）：儿童肿瘤组（COG）第1阶段和试点联盟的使命是通过合理有效的临床和实验室研究，为儿童和青少年癌症患者确定和开发有效的新药物。这个有限的机构联盟，由21个首屈一指的儿科肿瘤学项目组成，这些项目是通过同行评审过程选出的，是儿科肿瘤学新药开发的国家和国际模式。该联盟成功地利用了母公司儿童肿瘤学组（COG）的数据库基础设施和资源，同时维护自己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。该联盟拥有进行转化生物学、药代动力学和药物遗传学研究的专家资源，并开发了最先进的基础设施，以促进专门相关成像研究的图像传输，用于集中审查和分析。该联盟的主要具体目标是：1）基于来自临床前肿瘤模型系统、已知分子靶点和/或成人临床试验数据的可用数据，对靶向特定肿瘤类型的药剂进行I期试验; 2）与COG疾病委员会合作，根据肿瘤模型系统的现有数据，开发和开展针对特定类型癌症的有希望的新治疗方法的试点研究，肿瘤的分子特征和儿科1期或2期试验数据;以及3）将药代动力学和药效学终点（包括成像和/或转化实验室研究）酌情纳入1期和先导性研究，以促进新治疗方法的未来开发。儿童癌症的靶向治疗的发展是一个高度优先事项，因为这种治疗提供了更有效和毒性更低的前景。

DESCRIPTION (provided by applicant): The mission of the Children's Oncology Group (COG) Phase 1 and Pilot Consortium is to identify and develop effective new agents for children and adolescents with cancer, through rational and efficient clinical and laboratory research. This limited institution consortium, comprised of 21 premier pediatric oncology programs in the country that were selected through a peer review process, serves as a national and international model for new agent development in pediatric oncology. The Consortium successfully leverages the database infrastructure and resources of the parent Children's Oncology Group (COG) while maintaining its own administrative and operational infrastructure to ensure rapid development, implementation and reporting of specialized and complex early phase clinical trials. The Consortium has expert resources for the conduct of translational biology, pharmacokinetic, and pharmacogenetics studies, and has developed a state-of-the art infrastructure to facilitate image transfer of specialized correlative imaging studies for central review and analyses. The Consortium's primary specific aims are: 1) To perform phase 1 trials of agents that target specific tumor types based on available data from preclinical tumor model systems, known molecular targets, and/or adult clinical trial data; 2) To collaborate with COG Disease Committees to develop and conduct pilot studies of promising new treatment approaches with specific types of cancer, based on available data from tumor model systems, the molecular profile of the tumor, and pediatric phase 1 or 2 trial data; and 3) To incorporate pharmacokinetic and pharmacodynamic endpoints, including imaging and/or translational laboratory studies, as appropriate into Phase 1 and pilot studies to facilitate future development of the new treatment approaches. The development of targeted therapy for childhood cancers is a high priority as such therapeutics offer the prospect of being more efficacious and less toxic.