Enroll-HD platform support for clinical trials and studies in Huntington’s disease [Conference Abstract]

Townhill, Jenny, Bronzova, Juliana, Sathe, Swati and Sampaio, Cristina 2026. Enroll-HD platform support for clinical trials and studies in Huntington’s disease [Conference Abstract]. Journal of Neurology, Neurosurgery and Psychiatry 97 (Supp 2) , pp. A88-A89. 10.1136/jnnp-2026-ehdn.199

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Abstract

Background: Enroll-HD is a global, prospective, longitudinal observational study and research platform that supports Huntington’s disease (HD) clinical research and therapeutic development. Aim: To describe the Enroll-HD resources and infrastructure that support the planning and conduct of HD clinical studies. Methods: Enroll-HD provides the HD research community with access to comprehensive clinical datasets and biosamples. The platform offers scientific and operational support, including protocol design consultation, feasibility assessments, site identification, participant recruitment, and investigator training through its clinical training portal. Outcomes: Long-term management and monitoring of Enroll-HD and platform studies have established extensive expertise across more than 150 HD clinical sites, facilitating informed site selection for clinical research. Real-time, in silico ­screening of the Enroll-HD database enables estimation of potentially eligible participants using study-specific inclusion criteria. The Global Site Investigator Database (GSID) further supports site identification and selection for studies and is used to assess trial capabilities. Enroll-HD data are increasingly used as external comparator cohorts in interventional studies. The European Medicines Agency has qualified Enroll-HD as a data source and infrastructure for post-authorisation monitoring of HD therapies, and the U.S. Food and Drug Administration recognises it as a suitable source of natural history data for regulatory submissions. Several CHDI-sponsored studies, including HDClarity and LSA, use a nested study design in which Enroll-HD provides core assessment data and operational infrastructure, including electronic data capture and monitoring. Conclusions: Enroll-HD is a mature, regulatory-recognised platform that integrates datasets, biosamples, site networks, and operational capabilities to accelerate HD clinical research and therapeutic development.

Item Type:	Short Communication
Date Type:	Publication
Status:	Published
Schools:	Schools > Biosciences
Publisher:	BMJ Publishing Group
ISSN:	0022-3050
Last Modified:	27 Oct 2026 15:01
URI:	https://orca.cardiff.ac.uk/id/eprint/190465

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