Pediatric Phase 1/Pilot Consortium

儿科 1 期/试点联盟

• 批准号: 9118080
• 负责人: Brenda J. Weigel
• 金额: $ 318.12万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-08-08 至 2018-09-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9118080
• 关键词: 19 year old; Acute; Acute Lymphocytic Leukemia; Acute Myelocytic Leukemia; Adolescent; Adult; Adverse effects; Antineoplastic Agents; Biological Models; Biology; Central Nervous System Neoplasms; Child; Child Mortality; Childhood; Chronic; Clinical Pharmacology; Clinical Research; Clinical Trials; Complex; Country; Data; Databases; Development; Disease; Dose; Drug Kinetics; Drug Targeting; Ensure; Evaluation; Foundations; Future; Goals; Image; Infant; Institution; International; Laboratories; Laboratory Research; Laboratory Study; Malignant - descriptor; Malignant Childhood Neoplasm; Malignant Neoplasms; Mission; Modeling; Molecular Profiling; Molecular Target; Neuroblastoma; New Agents; North America; Outcome; Parents; Pediatric Neoplasm; Pediatric Oncology; Pediatric Oncology Group; Peer Review; Performance; Pharmaceutical Preparations; Pharmacodynamics; Pharmacogenetics; Phase; Phase I Clinical Trials; Pilot Projects; Process; Recurrence; Refractory; Reporting; Research Infrastructure; Resources; Role; Safety; Signal Pathway; Staging; Therapeutic; Toxic effect; Translating; antitumor effect; base; cancer therapy; cancer type; chemotherapeutic agent; cytotoxic; design; meetings; novel anticancer drug; oncology program; pre-clinical; sarcoma; targeted agent; targeted treatment; treatment strategy; tumor

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.

描述（由申请人提供）：通过合理有效的临床和实验室研究，儿童肿瘤学组（COG）和试点联盟的任务是为儿童和癌症的儿童和青少年确定和开发有效的新药物。这个有限的机构财团由该国21个主要的儿科肿瘤学计划组成，这些计划是通过同行审查过程而选择的，它是儿科肿瘤学新代理发展的国家和国际模型。该财团成功利用了父母儿童肿瘤学集团（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，以确保对专业和复杂的早期临床试验的快速发展，实施和报告。该财团拥有用于转化生物学，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，以促进专门的相关成像研究的图像转移，以进行中央审查和分析。财团的主要特定目的是：1）基于临床前肿瘤模型系统，已知分子靶标和/或成人临床试验数据的可用数据，对特定肿瘤类型进行针对特定肿瘤类型的试验； 2）与COG疾病委员会合作，根据肿瘤模型系统的可用数据，肿瘤的分子特征以及儿科1或2期试验数据，开发和进行针对特定类型癌症的有希望的新治疗方法的试点研究； 3）将适当的药代动力学和药效动力学终点（包括成像和/或翻译实验室研究）纳入第1阶段以及试点研究，以促进新治疗方法的未来开发。儿童癌症靶向疗法的发展是一个很高的优先事项，因为这种治疗方法提供了更有效和毒性更少的前景。