Core--Clinical

临床核心将支持基因治疗卓越计划的研究人员制定，执行和分析其临床基因转移试验，目标是 确保患者安全、促进研究实施和实现法规遵从性。在方案制定期间，临床核心将在临床方案准备过程中提供研究设计、生物统计和监管支持。临床核心将协助研究者准备提交给机构审查委员会、机构生物安全委员会、一般临床研究中心、NIH重组DNA咨询委员会、FDA和内部机构委员会的文件。核心人员还将协助研究者准备其方案，以提交给数据和安全性监查委员会（DSMB）。在实施方案之前，临床中心将在编写研究手册、病例报告表和相关研究文件、标准操作 与研究实施、统计分析计划和监查计划相关的程序。在试验执行期间，临床中心将为研究程序、标本采集和处理以及患者旅行安排提供后勤支持。临床核心将促进研究人员与华盛顿大学综合临床研究中心和基因与细胞治疗实验室之间的互动。临床中心将验证知情同意书，填写病例报告表，并协助数据管理。临床核心的一项关键职能是就每项临床方案与外部监查员签订合同，以确保研究按照研究方案进行。质量保证稽查员将确保所有试验严格按照机构和政府法规进行。的 临床核心还将提供药物临床试验质量管理规范方面的持续培训，并将协助研究者向机构委员会、NIH和FDA提交不良事件报告和年度报告。研究完成后，临床中心将协助数据管理和分析以及长期随访。

The Clinical Core will support investigators of the Program for Excellence in Gene Therapy with the formulation, execution, and analysis of their clinical gene transfer trials, with the goals of ensuring patient safety, facilitating study implementation, and achieving regulatory compliance. During protocol development, the Clinical Core will provide study design, biostatistical, and regulatory support in preparing the clinical protocol. The Clinical Core will assist investigators in preparing submissions to the Institutional Review Board, Institutional Biosafety Committee, General Clinical Research Center, the Recombinant DNA Advisory Committee of the NIH, the FDA, and internal institutional committees. The Core will also assist investigators in preparing their protocols for presentation to the Data and Safety Monitoring Board (DSMB). Prior to implementing the protocol the Clinical Core will play an active role in the preparation of study manuals, case report forms, and related study documents, standard operating procedures related to study conduct, statistical analysis plans, and monitoring plans. During execution of the trial the Clinical Core will provide logistical support for study procedures, specimen collections and processing, and patient travel arrangements. The Clinical Core will facilitate interactions between investigators and the UW General Clinical Research Center and the Gene and Cell Therapy Laboratory. The Clinical Core will verify informed consent, complete case report forms, and assist with data management. A key function of the Clinical Core will be to contract with external monitors for each of the clinical protocols to assure that studies are conducted in accordance with the study protocol. A quality assurance auditor will assure that all trials are conducted strictly in compliance with institutional and governmental regulations. The Clinical Core will also provide ongoing training in Good Clinical Practice, and will assist investigators with submission of adverse event reports and annual reports to the institutional committees, the NIH, and the FDA. Following study completion the Clinical Core will assist with data management and analysis and long-term follow-up.