ArticleMovement disorders : official journal of the Movement Disorder Society2022
Utility of the Huntington's Disease Prognostic Index Score for a Perimanifest Clinical Trial.
Article in Movement disorders : official journal of the Movement Disorder Society, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed, 8 citations in OpenAlex.
- Modeling Disease Progression and Placebo Response in Huntington Disease: Insights From Enroll-HD and GENERATION HD1 Cohorts.Neurology · 2025Trial
- Development and validation of the first needs-based quality of life measure for individuals with manifest Huntington's disease.Journal of neurology · 2026Article
- SPARCC: Semi-Parametric Robust Estimation in a Right-Censored Covariate Model.Journal of the American Statistical Association · 2026Article
- Unraveling progressive verbal memory deficits in Huntington's disease: insights from the LASSI-L.Frontiers in neurology · 2025Article
- Towards Standardizing Nomenclature in Huntington's Disease Research.Journal of Huntington's disease · 2024Review
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSubtle neurodegenerative motor and cognitive impairments accumulate over a prodromal period several years before clinical diagnosis of Huntington's disease (HD). The inclusion of prodromal individuals in therapeutic trials would facilitate testing of therapies early in the disease course and the development of treatments intended to prevent or delay disability.
objectivesWe evaluate the normalized prognostic index (PIN) score as a tool to select participants for a perimanifest trial. We explore anticipated PIN-based inclusion rates from the preHD screening population and estimate sample-size requirements based on PIN threshold, trial duration, and outcome measure.
methodsIndividual participant data from ENROLL-HD were used to fit mixed effect linear models to assess longitudinal changes in clinical metrics for participants with early-manifest HD and PIN-stratified preHD subcohorts.
resultsA PIN threshold of 0.0 was met by 40% of the preHD participants in ENROLL-HD; 39.4% and 55.2% progressed to new diagnoses of early-manifest HD within 2 and 3 years, respectively. Various PIN thresholds also enabled the selection of specified ratios of prodromal preHD to early manifest HD participants for a perimanifest trial. Estimated sample sizes for a trial enrolling prodromal preHD (PIN > 0.0) and stage 1 and 2 motor-diagnosed participants varied depending on the composition of the screening pool, the length of follow-up (1, 2, or 3 years), and outcome measure.
conclusionsThe composition of a perimanifest clinical trial population can be defined using preselected PIN thresholds, facilitating the assessment of potential disease-modifying therapies in HD. © 2022 Voyager Therapeutics, Inc. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson Movement Disorder Society.
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