A12 How does Enroll-HD expedite/facilitate the conduct of clinical trials?. (13th September 2016)

• Record Type: Journal Article
• Authors: McLean, Tim; Mestre, Tiago; Levey, Jamie; Handley, Olivia; Landwehrmeyer, Bernhard; Sampaio, Cristina
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 87(2016)Supplement 1
• Page Start: A4
• Page End: A4
• Publication Date: 2016-09-13
• Subjects: HD Clinical trials; Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2016-314597.12
• Languages: English
• ISSNs: 0022-3050

Abstract

Abstract : Background: In recent years there has been a significant increase in the number and size of clinical trials in HD, often competing for the same or similar patient population and HD clinical site resources. This trend is set to continue as clinical trials move into later phases of development. Enroll-HD is a global, prospective, longitudinal observational study of Huntington's disease subjects and forms the core of the Enroll-HD research platform. Aims: A key aim of the Enroll-HD study is to enhance the design and expedite the conduct of clinical trials in HD. Methods: The Enroll-HD study is so far active in over 140 HD clinical sites in North and South America, Europe, Australia and New Zealand and has recruited over 10, 900 participants including gene carriers (premanifest and manifest) as well as individuals at risk and controls. Assessments performed annually include a core data battery of motor, cognitive and behavioural symptoms in addition to the collection of socio-demographic information, medical history, co-morbid conditions and current therapies. CAG repeat values are recorded for research purposes. All participants provide written informed consent including an option to be contacted for possible future clinical research participation. Study-specific interrogation of the database enables the production of listings of potentially eligible clinical trial patients. The Enroll-HD data are monitored and sites supported by a network of regionally based HD specialist monitors and/or site managers who have longstanding relationships with the sites and are available to support study teams throughout all stages of their trials. The Enroll-HD research platform includes a developing clinical training portal intended to host training modules relevant to HD clinical trials for participating Enroll-HD site staff. Results: The Enroll-HD platform has so far supported 4 large global clinical trials as well as numerous smaller studies including the selection of approximately 100 HD clinical trial sites; supported the recruitment of over 800 HD patients and provided advice and troubleshooting as an integrated part of the clinical trial project management teams. A fully developed training portal will be available for future trials. Acknowledgements: Enroll-HD is a longitudinal observational study for Huntington's disease families intended to accelerate progress towards therapeutics; it is sponsored by CHDI Foundation, a nonprofit biomedical research organisation exclusively dedicated to developing therapeutics for HD. Enroll-HD would not be possible without the vital contribution of the research participants and their families