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

Hampton L LeonardCornelis BlauwendraatLynne KrohnFaraz FaghriHirotaka IwakiGlen FergusonAaron G Day-WilliamsDavid J StoneAndrew B SingletonMike A NallsZiv Gan Ornull null

Published in: Journal of medical genetics (2019)

Our data support the hypothesis that within genetically unmatched clinical trials, genetic heterogeneity could confound true therapeutic effects as expected. Clinical trials should undergo pretrial genetic adjustment or, at the minimum, post-trial adjustment and analysis for failed trials.

Keyphrases

clinical trial
phase ii
phase iii
genome wide
study protocol
open label
copy number
double blind
single cell
dna methylation
big data
type diabetes
electronic health record
risk assessment
machine learning
skeletal muscle
climate change