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指导。该核心将建立在全国儿童医院 (NCH) 研究所神经肌肉临床研究计划的现有专业知识之上。 PI Mendell 博士已经组建的转化研究人员在设计和实施方面拥有丰富的专业知识 尖端临床研究项目。工作人员协调大型多中心协作项目的经验与 Weilstone MDCRC 的管理直接相关，包括协调多个站点之间的沟通的经验；质量控制和文件跟踪；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。联合肌营养不良项目将促进接触患者，该项目是一个由 NIH 资助的大型多中心基因型/表型和自然史联盟，由最近加入 NCH 基因治疗中心的 Kevin Flanigan 博士领导。 Mendell博士在DMD临床试验的设计和实施方面拥有丰富的经验，包括在基因转移试验的实施方面拥有独特的经验，他和Flanigan博士都曾担任目前正在进行的基因转移试验或基因校正（外显子跳跃和无义抑制）试验的设计顾问。 核心将负责在 NCH MDCRC 内部和更广泛的 Weilstone 网络之间建立和维持调查人员之间的有效沟通与合作。在此角色中，它将制定明确的内部和外部程序来监测和评估研究项目和核心设施；建立研究者之间管理和共享数据、动物模型和其他资源的机制；为校外资助和行业资助的转化研究项目的发展以及 MDCRC 的教育核心活动提供行政支持。 行政核心还将设立一个中心咨询委员会，并制定框架以确保该委员会为监督中心的研究和翻译活动做出贡献。