I30 Enroll-HD: a global clinical research platform for huntington's disease. (13th September 2016)

• Record Type: Journal Article
• Authors: Handley, Olivia; Mestre, Tiago; Levey, Jamie; McLean, Tim; Landwehrmeyer, Bernhard; Sampaio, Cristina
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 87(2016)Supplement 1
• Page Start: A69
• Page End: A69
• Publication Date: 2016-09-13
• Subjects: Clinical research platform; Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2016-314597.195
• Languages: English
• ISSNs: 0022-3050

Abstract

Abstract : Background: Enroll-HD is not only a study of HD; it serves as a global research platform to expedite clinical trials, understand more about the disease, and improve care. Input from clinical sites, HD families, Enroll-HD governance and lay associations has enabled a dynamic infrastructure to support and develop a host of platform activities. Aims: The Enroll-HD study is at the core of the research platform. Its clinical and biological repository of over 10, 000 participants provides a rich and valuable scientific resource. Together with its established network of clinical research sites, the Enroll-HD platform is able to continue its focus on the following key aims: to accelerate the discovery and development of new therapeutics for HD, to inform our understanding of the disease, and to lead to improvements in care. Methods/techniques: Enroll-HD has a network of 136 clinical research sites. Clinical and biological samples are collected annually according to a core assessment battery. Following quality control review, data and samples are made available for wider use in the research community. Results/outcome: The Enroll-HD platform has enabled a series of outputs for the research community: Access to data and samples: >70 approved applications for datasets and five approved requests for biosamples. Platform studies: Enroll-HD offers a range of services that can be leveraged to support platform studies, e.g., site/participant feasibility, EDC implementation, study start-up, study management and data extraction. Clinical trials- feasibility: With a clinical research network of >130 sites, the Enroll-HD Operations team supports sites feasibility and selection for upcoming clinical trials, as well as supporting population sample feasibility. Training: The Enroll-HD Clinical Training Portal hosts training materials relevant to HD clinical trials and studies. Conclusions: The platform has seen a series of successful outcomes made possible only by the collaboration of HD families, clinical and scientific research staff, lay organisations and the Enroll-HD governance committees. 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.