M02 Enroll-HD: a prospective observational study in a global huntington's disease cohort

Background Enroll-HD is a global, observational, longitudinal study that combines two existing Huntington9s disease (HD) observational studies, “REGISTRY” conducted in Europe and “COHORT” in North America and Australia. Enroll-HD will also include new sites in Latin America and Asia to establish one comprehensive observational study collecting a common data set across all sites. Aims The objective of Enroll-HD is to accelerate the development of therapies for HD by (1) compiling uniform clinical data and biological samples critical to better understand the natural history of HD, (2) building a comprehensive database and catalogue of bio-samples that will be available for use by qualified HD researchers worldwide, (3) making available larger datasets from a greater number of research participants, thereby enhancing the power of data-mining and computational biology projects, (4) serving as a platform to facilitate clinical sub-studies and the development and validation of novel assessment tools for HD, and (5) expediting recruitment into global clinical trials of candidate therapeutics in the coming years. Methods A collaborative process for gathering input from global stakeholders was implemented using nine working groups involving 95 participants from 19 countries. The effort was led by an Executive Oversight Committee and managed by a team of project management professionals. The working groups each focused on a specific and critical phase of the start-up process, including, protocol development, data privacy, ethics, bio-repositories, etc. This process has been ongoing to address issues, plan implementation and ensure compliance. Results The protocol was approved September 2011. The first site IRB-approval was received April 2012.The Authors will provide an update on the start-up and implementation of the study in North America and present the plans for ongoing implementation across Latin America, Europe, and Australasia. In addition, details of the Enroll-HD governance structure will be presented. Conclusions With its broad and global reach, Enroll-HD will not only build upon existing strong collaborative relationships among HD researchers, but also create a new, international platform for HD clinical research which will help to accelerate future clinical trials.