Genetic variability and potential effects on clinical trial outcomes: perspectives in Parkinson's disease.

ABSTRACT:

Background

Classical randomisation of clinical trial patients creates a source of genetic variance that may be contributing to the high failure rate seen in neurodegenerative disease trials. Our objective was to quantify genetic difference between randomised trial arms and determine how imbalance can affect trial outcomes.

Methods

5851 patients with Parkinson's disease of European ancestry data and two simulated virtual cohorts based on public data were used. Data were resampled at different sizes for 1000 iterations and randomly assigned to the two arms of a simulated trial. False-negative and false-positive rates were estimated using simulated clinical trials, and per cent difference in genetic risk score (GRS) and allele frequency was calculated to quantify variance betwe

SUBMITTER: Leonard H 

PROVIDER: S-EPMC8474559 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature