K008 Enroll-HD study status

<h3></h3> Enroll-HD is a clinical research platform that includes, at its core, an observational, prospective study of Huntington’s disease (HD). The objectives are to 1) expedite the conduct of clinical trials, 2) improve the understanding of HD, and 3) foster good clinical care. As of May 1 2024, 31,622 participants have been recruited from 189 sites (of which 155 are active) in 23 countries, of which 21,605 are still current (i.e., no mortality/end form) and 16.512 are active (i.e., have not missed two or more visits). Re-coded data and biosamples are freely available to researchers and have already underpinned significant scientific breakthroughs. The sixth periodic dataset was released in January 2023 and contains data from 25,550 participants and 95,040 visits. The Functional Rating Scale 2.0 (FuRST 2.0) and a Sleep Assessment were added to Enroll-HD’s extended assessment battery at sites in the United States and the United Kingdom and will be available to other countries in the future. The longitudinal collection of RNA has recently been initiated. An amendment to the protocol (Enroll-HD 2.0) is underway, involving a transition from a broad recruitment approach to targeted recruitment based on the Huntington’s Disease Integrated Staging System, and specific assessment batteries tailored according to disease stage. An increasing number of clinical trials/studies are utilizing at least one area of the Enroll-HD platform support, including site feasibility, guidance on study design, potentially eligible participant listings, study set-up support, monitoring, and data management. The Enroll-HD Clinical Training Portal offers online training for the Unified Huntington’s Disease Rating Scale Motor Certification (all users), FuRST 2.0 (Enroll-HD users), and Enroll-HD RNA and Plasma Collection (Enroll-HD users). Training modules also support other studies hosted by the platform. The portal successfully enables faster, cost-effective standardization of training.