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

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.

Keywords: Enroll-HD; Huntington's disease; clinical research platform; disease network; longitudinal observational cohort study; registry.

Figure 1

Schematic outlining the Enroll-HD clinical research platform.

Figure 2

Number of participants from Enroll-HD alone and in combination with precursor studies like REGISTRY with specified number of visits (N = 21,116).

Figure 3

Geographical distribution of Enroll-HD participants (N = 21,116).

Figure 4

Participant category at Enroll-HD baseline visit (N = 21,116).