Clinical Translational Core

RFA-HD-13-002要求在每个IDDRC中包含一个临床翻译核心， 处理一系列活动，包括：(A)招募研究参与者，(B)主题 评估、表型鉴定或治疗；(C)生物标志物的开发；(D)制造服务；(E)高生产能力 筛查和/或(F)从IDD患者身上收集样本以用于生物库。如中所述 我们的介绍性概述和以下核心描述，这些活动中的许多都得到了其他 我们开发的大型多学科研究环境中的核心和现有资源 多年来的各种机构。因此，临床翻译核心的总体目标是补充 这些资源并协调国际发展研究中心支持的调查人员的准入。这将有助于我们的初选 加强将基础科学发现转化为有效的、商业上可用的改变疾病的目标 为遗传性和后天智力和发育障碍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)，这是一个建立了三年的计划 为了回应我们IDDRC内部和更广泛的领域对翻译研究的日益重视 它所支持的研究计划。CTU专注于针对发育障碍的治疗方法的开发 精神障碍，包括在所有阶段进行调查性dmg试验。临床翻译核心意志 扩大CTU的能力，提供专家资源，包括生物统计和方案咨询 向机构审查委员会(IRB)提交方案之前的设计、预审和实际操作建议 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。

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.