Children's Center for Clinical Pharmacology Studies

描述（由申请人提供）： 本提案是对RFA #HD-01-003的回应，旨在继续资助位于密苏里州堪萨斯城的儿童慈善医院和诊所（CMH）的儿科药理学研究单位，作为PPRU的NICHD网络内的研究中心之一。该单位的总体目标是：1）继续并扩大CMH PPRU在PPRU网络中的合作参与，主要目的是通过创建新的信息和知识来生成支持儿童药物标签的数据;和2）在发育药理学方面进行基础和转化研究，这将促进我们对遗传-年龄-和疾病-药物处置和作用的相关差异（有利和不利）。具体目标包括：1）合作进行新药和目前上市的药物的研究，优先考虑PPRU网络研究; 2）识别、整理、分析和解释来自儿科临床试验的数据，这些数据足以扩展儿科标签; 3）使用分子方法来定义儿童发育、疾病表达、药物生物转化和药物作用之间的“联系”; 4）运用生物信息学、药物基因组学、代谢组学等学科知识，探索和明确重要药物代谢酶和转运蛋白的发育调控基础; 5）提供独特的教学环境，使儿科医生、医学专科医生、临床药师、护士和专职医疗人员能够获得临床研究和儿科临床药理学方面的专业知识。研究者和申请机构实现这些目标和具体目标的能力通过以下方式得到证明：I）有效的计划组成和管理; 2）申请机构内部的专业知识和资源; 3）与PPRU网络相关的先前和当前研究成果; 4）预计计划扩展，以满足未来五年对儿科临床试验预期增加的需求，加强转化科学与药物开发过程的整合;以及5）提出两项新的研究提案，这些提案直接涉及计划目标，非常适合作为PPRU网络的一部分进行。

DESCRIPTION (provided by applicant): This proposal is a sent in response to RFA #HD-01-003 to continue funding of a Pediatric Pharmacology Research Unit at Children's Mercy Hospitals and Clinics (CMH) in Kansas City, Missouri as one of the sites within the NICHD Network of PPRU's. The overall objectives of the Unit are: 1) to continue and expand collaborative participation of the CMH PPRU in the PPRU Network for the primary purpose of producing data that will support labeling of drugs for children through the creation of new information and knowledge; and 2) To conduct basic and translational research in developmental pharmacology that will advance our understanding of the impact of genetic- age- and disease-related differences in drug disposition and action (both beneficial and adverse). Specific Aims include: 1) The collaborative conduct of studies of new and currently marketed drugs with priority given to PPRU Network studies; 2) The identification, collation, analysis, and interpretation of data from pediatric clinical trials that are sufficient for expansion of pediatric labeling; 3) Use of molecular approaches to define the "link" between childhood development, disease expression, drug biotransformation and drug action; 4) Use of bioinformatics, pharmacogenomics, metabolomics to explore and define the basis of developmental regulation of important drug metabolizing enzymes and transporters; 5) The provision of a unique teaching environment in which pediatricians, medical sub-specialists, clinical pharmacists, nurses and allied health care professionals can gain expertise in clinical investigation and the discipline of pediatric clinical pharmacology. The capability of the investigators and applicant institution to accomplish these objectives and specific aims is demonstrated by: I) effective programmatic composition and administration; 2) expertise and resources within the applicant institution; 3) previous and current research accomplishments related to the PPRU Network; 4) projected programmatic expansion necessary to meet the anticipated increased demands for pediatric clinical trials over the next five years and also, to enhance the integration of translational science into the drug development process; and 5) presentation of two new research proposals which speak directly to the program objectives and are ideally suited for conduct as part of the PPRU Network.