Robust Markers and Sample Sizes for Multicenter Trials of Huntington Disease.

ABSTRACT:

Objective

The identification of sensitive biomarkers is essential to validate therapeutics for Huntington disease (HD). We directly compare structural imaging markers across the largest collective imaging HD dataset to identify a set of imaging markers robust to multicenter variation and to derive upper estimates on sample sizes for clinical trials in HD.

Methods

We used 1 postprocessing pipeline to retrospectively analyze T1-weighted magnetic resonance imaging (MRI) scans from 624 participants at 3 time points, from the PREDICT-HD, TRACK-HD, and IMAGE-HD studies. We used mixed effects models to adjust regional brain volumes for covariates, calculate effect sizes, and simulate possible treatment effects in disease-affected anatomical regions. We used our model to estimate th

SUBMITTER: Wijeratne PA 

PROVIDER: S-EPMC7187160 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature