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.
Highlights
Enroll-Huntington’s disease (HD) is a worldwide integrated clinical research platform that has, at its core, an observational study that has recruited almost 25,000 participants
Enroll-HD, Research Platform, and Observational Study dedicated to collaboratively developing therapeutics that will substantially improve the lives of those affected by HD
Studies built into the platform and that recruit participants within the large Enroll-HD cohort and that involve additional assessments are referred to as nested studies; currently, these studies are investigating biomarkers and clinical or patient-reported outcomes
Summary
Enroll-HD is a worldwide integrated clinical research platform that has, at its core, an observational study that has recruited almost 25,000 participants. The platform was designed as a low-burden entry for participants into HD research, and provides a large, wellcharacterized, research-engaged cohort with associated clinical data and biosamples to facilitate recruitment into interventional trials and other research studies. Central to the clinical research platform is the Enroll-HD study (NCT01574053), a prospective longitudinal observational study that collects natural history data in HD gene-expansion carriers (HDGECs) and non-HDGEC controls. Established in July 2012, the Enroll-HD study has generated a large and rich clinical database with associated biosamples to support research, including developing disease-progression and prognostic biomarkers, identifying clinically relevant phenotypic characteristics, and establishing endpoints for interventional trials. There is an emphasis on recruiting premanifest HDGEC participants into Enroll-HD, especially younger adults, to identify early biological and clinical characteristics with a view to designing clinical trials earlier in the disease course. The study population comprises HDGECs (CAG expansion ≥36 on the longer allele) classified as: 1. Manifest HD: HDGECs age 18 or older who are deemed to have diagnostic HD clinical features in the opinion of the site investigator (and confirmed at each subsequent visit)
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