Utility of the Huntington's Disease Prognostic Index Score for a Perimanifest Clinical Trial.

ABSTRACT:

Background

Subtle 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.

Objectives

We 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.

Methods

Individual participant data from ENROLL-HD were used to fit mixed effect linear model

SUBMITTER: Langbehn DR 

PROVIDER: S-EPMC9306552 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature