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I30 Enroll-HD: a global clinical research platform for huntington’s disease
  1. Olivia Handley1,
  2. Tiago Mestre2,
  3. Jamie Levey3,
  4. Tim McLean1,
  5. Bernhard Landwehrmeyer4,
  6. Cristina Sampaio3 on behalf of the Enroll-HD Steering Committee
  1. 1EHDN, University of Ulm, Germany
  2. 2University of Ottawa, Canada
  3. 3CHDI Management/Foundation, Inc., Princeton, NJ, USA
  4. 4University of Ulm, Germany

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.

  • Clinical research platform

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