Reproducibility of cognitive endpoints in clinical trials: lessons from neurofibromatosis type 1.

Jonathan M PayneStephen J C HearpsKarin S WalshIris PaltinBelinda BartonNicole J UllrichKristina M HaebichDavid CoghillGerard A GioiaAlan CantorGary CutterJames H TonsgardDavid ViskochilCeliane Rey-CasserlyElizabeth K SchorryJoseph D AckersonLaura KlesseMichael J FisherDavid H GutmannTena RosserRoger J PackerBruce KorfMaria T AcostaKathryn N Northnull null

Published in: Annals of clinical and translational neurology (2019)

These data demonstrate that single observed endpoints are not appropriate to determine efficacy, partly accounting for the poor test-retest reliability of cognitive outcomes in clinical trials in neurodevelopmental disorders. Recommendations to improve reproducibility are outlined to guide future trial design.

Keyphrases

clinical trial
phase ii
phase iii
study protocol
open label
electronic health record
double blind
current status
big data
clinical practice
randomized controlled trial
metabolic syndrome