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
Statistics from Altmetric.com
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.