Pediatric Phase 1/Pilot Consortium

儿科 1 期/试点联盟

基本信息

批准号：
8580191
负责人：
Brenda J. Weigel
金额：
$ 213.14万
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：
2002
资助国家：
美国
起止时间：
2002-08-08 至 2017-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8580191
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 Children&apos s Oncology Group 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 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 base cancer therapy cancer type chemotherapeutic agent cytotoxic design meetings novel oncology program pre-clinical sarcoma treatment strategy tumor

项目摘要

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）：儿童肿瘤学组（COG）第一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，为患有癌症的儿童和青少年识别和开发有效的新药。这个有限的机构联盟由通过同行评审过程选出的国内 21 个主要儿科肿瘤学项目组成，成为儿科肿瘤学新药物开发的国家和国际典范。该联盟成功地利用母公司儿童肿瘤学组 (COG) 的数据库基础设施和资源，同时维护自己的管理和运营基础设施，以确保快速开发、实施和报告专门且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，以供中央审查和分析。该联盟的主要具体目标是： 1) 根据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，对针对特定肿瘤类型的药物进行一期试验； 2) 与 COG 疾病委员会合作，根据肿瘤模型系统的可用数据、肿瘤的分子概况以及儿科 1 期或 2 期试验数据，开发和开展针对特定类型癌症的有前景的新治疗方法的试点研究； 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）酌情纳入第一阶段和试点研究，以促进新治疗方法的未来开发。儿童癌症靶向治疗的开发是当务之急，因为此类疗法有望更有效且毒性更小。