CORE--GENE THERAPY WORKING GROUP/BIOSTATISTICS

核心——基因治疗工作组/生物统计学

基本信息

批准号：
6194936
负责人：
James M Croop
金额：
$ 12.08万
依托单位：
INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS
依托单位国家：
美国
项目类别：
财政年份：
1999
资助国家：
美国
起止时间：
1999-09-15 至 2000-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/6194936
关键词：
biomedical facility blood disorder clinical research clinical trials experimental designs gene therapy health science research analysis /evaluation statistics /biometry

项目摘要

The Gene Therapy Working Group and Biostatistics Core provides the services necessary for the development, administration and evaluation of gene therapy clinical trials. This core is a unique multi-disciplinary group that brings together the principal participants of gene therapy clinical trials in an ongoing weekly meeting. The Gene Therapy Working Group and Biostatistics Core has been the focal point for coordination of preclinical development, protocol inception, regulatory approval, protocol execution, and data retrieval and analysis. The use of this core is expected to increase over the next few years as patient trials are pursued. This core provides direction for preclinical basic science investigations to generate the information necessary to rationally design gene therapy clinical trials. This has resulted in a collaborative effort in construction of retroviral vectors and in defining preclinical and clinical parameters for stem cell manipulation. The specific service component of the Gene Therapy Working Group and Biostatistics Core is coordination of protocol development, regulation approval, interfacing clinical care with research components of the trial, and management of clinical data. These services are implement by the Gene Therapy Clinical Research Coordinator and Gene Therapy Research Nurse. The Gene Therapy Working Group and Biostatistics Core provides the organization and coordination required for these complex initiatives which demand the interaction of individuals with a wide range of expertise performing distinct roles. The core provides an environment for ongoing analysis and quality control of clinical trials. The integration of these practical components represents the cornerstone for efficient, successful development and execution of clinical gene therapy protocols.

基因治疗工作组和生物统计学核心为基因治疗临床试验的开发、管理和评估提供必要的服务。该核心是一个独特的多学科小组，将基因治疗临床试验的主要参与者聚集在一起，每周举行一次会议。基因治疗工作组和生物统计核心一直是协调临床前开发、方案启动、监管批准、方案执行以及数据检索和分析的焦点。随着患者试验的进行，预计未来几年该核心的使用将会增加。该核心为临床前基础科学研究提供方向，以生成合理设计基因治疗临床试验所需的信息。这导致了在构建逆转录病毒载体以及定义干细胞操作的临床前和临床参数方面的合作。基因治疗工作组和生物统计核心的具体服务部分是协调方案开发、法规批准、临床护理与试验研究部分的衔接以及临床数据的管理。这些服务由基因治疗临床研究协调员和基因治疗研究护士实施。基因治疗工作组和生物统计核心提供了这些复杂举措所需的组织和协调，这些举措需要具有广泛专业知识、扮演不同角色的个人进行互动。该核心为临床试验的持续分析和质量控制提供了环境。这些实用组件的整合是高效、成功开发和执行临床基因治疗方案的基石。