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Enroll-HD:一个用于亨廷顿舞蹈症的综合临床研究平台及全球观察性研究。

Enroll-HD: An Integrated Clinical Research Platform and Worldwide Observational Study for Huntington's Disease.

作者信息

Sathe Swati, Ware Jen, Levey Jamie, Neacy Eileen, Blumenstein Robi, Noble Simon, Mühlbäck Alzbeta, Rosser Anne, Landwehrmeyer G Bernhard, Sampaio Cristina

机构信息

CHDI Management/CHDI Foundation, Princeton, NJ, United States.

Department of Neurology, Ulm University, Ulm, Germany.

出版信息

Front Neurol. 2021 Aug 18;12:667420. doi: 10.3389/fneur.2021.667420. eCollection 2021.

Abstract

Established in July 2012, Enroll-HD is both an integrated clinical research platform and a worldwide observational study designed to meet the clinical research requirements necessary to develop therapeutics for Huntington's disease (HD). The platform offers participants a low-burden entry into HD research, providing a large, well-characterized, research-engaged cohort with associated clinical data and biosamples that facilitates recruitment into interventional trials and other research studies. Additional studies that use Enroll-HD data and/or biosamples are built into the platform to further research on biomarkers and outcome measures. Enroll-HD is now operating worldwide in 21 countries at 159 clinical sites across four continents-Europe, North America, Latin America, and Australasia-and has recruited almost 25,000 participants, generating a large, rich clinical database with associated biosamples to expedite HD research; any researcher at a verifiable research organization can access the clinical datasets and biosamples from Enroll-HD and nested studies. Important operational features of Enroll-HD include a strong emphasis on standardization, data quality, and protecting participant identity, a single worldwide study protocol, a flexible EDC system capable of integrating multiple studies, a comprehensive monitoring infrastructure, an online portal to train and certify site personnel, and standardized study documents including informed consent forms and contractual agreements.

摘要

“亨廷顿病登记研究(Enroll-HD)”成立于2012年7月,它既是一个综合临床研究平台,也是一项全球性观察性研究,旨在满足开发亨廷顿病(HD)治疗方法所需的临床研究要求。该平台为参与者提供了低负担参与HD研究的途径,提供了一个规模大、特征明确、参与研究的队列,并伴有相关临床数据和生物样本,便于招募进入干预试验和其他研究。利用Enroll-HD数据和/或生物样本的其他研究已纳入该平台,以进一步研究生物标志物和结局指标。Enroll-HD目前在全球21个国家的四大洲(欧洲、北美、拉丁美洲和澳大拉西亚)的159个临床地点开展业务,已招募了近25000名参与者,生成了一个庞大、丰富的临床数据库及相关生物样本,以加速HD研究;任何在可核实研究机构工作的研究人员都可以访问Enroll-HD及嵌套研究的临床数据集和生物样本。Enroll-HD的重要运营特点包括高度重视标准化、数据质量和保护参与者身份,采用单一的全球研究方案,具备整合多项研究能力的灵活电子数据采集(EDC)系统,全面的监测基础设施,用于培训和认证现场人员的在线门户,以及包括知情同意书和合同协议在内的标准化研究文件。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e34d/8416308/8118ede71374/fneur-12-667420-g0001.jpg

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