Administrative Core

行政核心

• 批准号: 8032763
• 负责人: Jerry Roy Mendell
• 金额: $ 9.17万
• 依托单位: RESEARCH INST NATIONWIDE CHILDREN'S HOSP
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-09-01 至 2015-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8032763
• 关键词: Accountability; Advisory Committees; Animal Model; Center for Translational Science Activities; Clinical; Clinical Research; Clinical Trials; Clinical Trials Design; Communication; Core Facility; Development; Duchenne muscular dystrophy; Ensure; Exons; Extramural Activities; Funding; Gene Transfer; Genotype; Industry; Monitor; Natural History; Patients; Pediatric Hospitals; Performance; Phenotype; Procedures; Quality Control; Reporting; Research Institute; Research Personnel; Research Project Grants; Resources; Role; Site; Translational Research; data management; data mining; data sharing; design; experience; gene correction; gene therapy; neuromuscular; programs

The Administrative Core (J. Mendell, PI) will provide scientific and fiscal oversight of and direction to the MDCRC Program. The Core will build upon the existing expertise of the Neuromuscular Clinical Research Program at the Research Institute at Nationwide Children's Hospital (NCH). The translational research staff already assembled by the PI, Dr. Mendell, has a demonstrated expertise in designing and implementing cutting-edge clinical research projects. The staff's experience in coordinating large multi-center collaborative programs is directly relevant to administration of a Weilstone MDCRC, and includes experience in coordination of communication among multiple sites; quality control and document tracking; data management; data mining and reporting; milestone and task management; and fiscal accountability. Access to patients will be facilitated by the United Dystrophinopathy Project, a large NIH-funded multicenter genotype/phenotype and natural history consortium directed by Dr. Kevin Flanigan, who has recently joined the NCH Center for Gene Therapy. Dr. Mendell has extensive experience in the design and performance of DMD clinical trials, including unique experience in the performance of gene transfer trials, and both he and Dr. Flanigan have served as consultants for the design of trials of gene transfer or gene correction (exon skipping and nonsense suppression) trials that are currently underway. The Core will be responsible for establishing and maintaining effective communications and cooperation among investigators both inside the NCH MDCRC and among the wider Weilstone network. In this role, it will develop clear internal and external procedures for monitoring and evaluating the research projects and core facilities; establish mechanisms for managing and sharing data, animal models, and other resources among investigators; and provide administrative support for the development of both extramural funded and industry-sponsored translational research projects, and for the Educational Core activities of the MDCRC. The Administrative Core will also establish a Center Advisory Committee, and develop the framework to ensure the contribution of this Committee to the oversight of the Center's research and translational activities.

行政核心（J. Mendell，PI）将为MDCRC计划提供科学和财政监督和方向。核心将基于全国儿童医院（NCH）研究所神经肌肉临床研究计划的现有专业知识。 PI已经组装的翻译研究人员Mendell博士在设计和实施方面具有专业知识 尖端的临床研究项目。员工协调大型多中心协作计划的经验与Weilstone MDCRC的管理直接相关，并包括在多个站点之间协调沟通的经验；质量控制和文档跟踪；数据管理；数据挖掘和报告；里程碑和任务管理；和财政问责制。联合营养不良项目将促进患者的访问，这是由NIH资助的大型多中心基因型/表型和自然历史联盟（Kevin Flanigan）博士指导的，他最近加入了NCH基因疗法中心。 Mendell博士在DMD临床试验的设计和性能方面拥有丰富的经验，包括在基因转移试验的性能方面的独特经验，而He和Flanigan博士曾是目前正在进行的基因转移或基因校正试验（外显子跳过和废话抑制）试验的顾问。 核心将负责建立和维持NCH MDCRC和更广泛的Weilstone网络中调查人员之间的有效沟通和合作。在此角色中，它将开发出明确的内部和外部程序，以监视和评估研究项目和核心设施；建立调查人员之间管理和共享数据，动物模型和其他资源的机制；并为开发由校外资助和行业赞助的翻译研究项目以及MDCRC的教育核心活动提供行政支持。 行政核心还将建立一个中心咨询委员会，并制定框架，以确保该委员会对中心研究和翻译活动的监督做出贡献。