Clinical Translational Core

临床转化核心

• 批准号: 8931784
• 负责人: MICHAEL Van Doren JOHNSTON
• 金额: $ 17.79万
• 依托单位: HUGO W. MOSER RES INST KENNEDY KRIEGER
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-23 至 2019-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8931784
• 关键词: Address; Adult; Basic Science; Biological Markers; Biometry; Child; Clinical; Clinical Trials; Clinical Trials Unit; Complement; Consultations; Development; Disease; Environment; Faculty; Genetic; Goals; Individual; Institution; Institutional Review Boards; Intellectual functioning disability; Interdisciplinary Study; Mental Retardation and Developmental Disabilities Research Centers; Participant; Phase; Phenotype; Protocols documentation; Research; Research Personnel; Resources; Services; Structure; Translational Research; biobank; design; developmental disease; experience; programs; response; sample collection; screening; therapy development

RFA-HD-13-002 requires the inclusion of a Clinical Translational Core within each IDDRC, addressing a combination of activities that includes: (a) recruitment of research participants, (b) subject assessment, phenotyping, or treatment, (c) development of biomarkers, (d) manufacturing services, (e) highthroughput screening, and/or (f) collection of samples from individuals with IDD for biobanking. As described in our Introductory Overview and the following Core descriptions, many of these activities are supported by other Cores and existing resources within the large and multidisciplinary research environment developed within our Institutions over the years. Therefore, the overall objective of the Clinical Translational Core is to complement those resources and coordinate access for IDDRC-supported investigators. This will contribute to our primary goal of enhancing the conversion of basic science discoveries into effective, commercially available disease-altering therapies for children and adults with genetic and acquired intellectual and developmental disabilities. The Core will be structured to complement our Clinical Trials Unit (CTU), a program established three years ago in response to the increasing emphasis on translational research within our IDDRC and the broader research program it supports. The CTU is focused on the development of treatments targeting developmental disorders, including conducting investigational dmg trials at all phases. The Clinical Translational Core will expand upon CTU capabilities to provide expert resources including consultation on biostatistics and protocol design, pre-review prior to submission of protocols to the institutional review board (IRB) and hands-on advice and help with carrying out investigational protocols by experienced clinical trials faculty, research coordinators and administrative staff.

RFA-HD-13-002要求在每个IDDRC中纳入临床翻译核心， 处理一系列活动，包括：（a）招募研究参与者，（B）研究对象 评估、表型分析或治疗，（c）生物标志物的开发，（d）制造服务，（e）高通量 筛选，和/或（f）从IDD个体收集样品用于生物库。中所述 我们的介绍性概述和以下核心描述，其中许多活动得到了其他 核心和现有资源的大型和多学科的研究环境中开发的，在我们的 机构多年来。因此，临床翻译核心的总体目标是补充 这些资源，并协调获得IDDRC支助的调查人员的访问。这将有助于我们的主要 目标是促进基础科学发现转化为有效的、商业上可获得的改变疾病的 为患有遗传性和后天性智力和发育残疾的儿童和成人提供治疗。 核心将被构建为补充我们的临床试验单位（CTU），一个成立三年的计划 为了应对我们的IDDRC和更广泛的翻译研究越来越重视 它支持的研究项目。CTU专注于开发针对发育障碍的治疗方法。 疾病，包括在所有阶段进行研究性DMG试验。临床翻译核心将 扩大反恐组的能力，提供专家资源，包括生物统计学和议定书方面的咨询 设计、向机构审查委员会（IRB）提交方案之前的预审查和实践建议 并由经验丰富的临床试验教师、研究协调员帮助执行研究方案 和行政人员。