Sample enrichment for clinical trials to show delay of onset in huntington disease.

ABSTRACT:

Background

Disease-modifying clinical trials in persons without symptoms are often limited in methods to assess the impact associated with experimental therapeutics. This study suggests sample enrichment approaches to facilitate preventive trials to delay disease onset in individuals with the dominant gene for Huntington disease.

Methods

Using published onset prediction indexes, we conducted the receiver operating curve analysis for diagnosis within a 3-year clinical trial time frame. We determined optimal cut points on the indexes for participant recruitment and then conducted sample size and power calculations to detect varying effect sizes for treatment efficacy in reducing 3-year rates of disease onset (or diagnosis).

Results

Area under the curve for 3 onset predi

SUBMITTER: Paulsen JS 

PROVIDER: S-EPMC8121118 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature