Year: 2024
Type: conference-paper
Abstract: Background Enroll-HD was designed as an observational, longitudinal study to describe the natural history of Huntington’s disease (HD) and support further research. The study has generated a vast amount of data and bio-samples which have been used in multiple analyses that have facilitated understanding of HD progression and variability and aided in identification of novel drug targets. Aims The amendment to the Enroll-HD protocol (referred to as ‘Enroll-HD 2.0’) aims to update the study to better reflect current scientific needs and support the next generation of clinical trials and research. Methods The development of the amendment included a coordinating/writing team and multiple consultations with stakeholders. The final documentation (i.e., protocol, manuals, and sub-study protocols) are being professionally reviewed for consistency and clarity. Results The stated objectives remain unchanged. The amended protocol changes the study’s design to put greater focus on early HD and use the HD-ISS to guide recruitment. Key changes include: • Participants will be assigned to one of several cohorts, which allows for assessments and procedures to be tailored to disease stage. • Shift away from an unspecified broad recruitment approach towards targeting specific populations using the HD-ISS and aim for an overall size of ~25,000 active participants. • Flexibility to collect additional bio-samples. • Sub-studies (including imaging) will be available at some sites for some participants. Conclusions Updating Enroll-HD is essential in order to optimize the platform’s ability to support clinical research. The amended protocol will be distributed at Enroll-HD Congress 2024. Site start-up activities are scheduled to begin in 2025.
Institutions: CHDI Foundation, European Huntington's Disease Network
Language: English
Subfield: Cellular and Molecular Neuroscience
Field: Neuroscience
Domain: Life Sciences
Open Access status: gold
OpenAlex ID: W4402374375