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

ABSTRACT:

Objective

Rapid developments in understanding the molecular mechanisms underlying cognitive deficits in neurodevelopmental disorders have increased expectations for targeted, mechanism-based treatments. However, translation from preclinical models to human clinical trials has proven challenging. Poor reproducibility of cognitive endpoints may provide one explanation for this finding. We examined the suitability of cognitive outcomes for clinical trials in children with neurofibromatosis type 1 (NF1) by examining test-retest reliability of the measures and the application of data reduction techniques to improve reproducibility.

Methods

Data were analyzed from the STARS clinical trial (n = 146), a multi-center double-blind placebo-controlled phase II trial of lovastatin, condu

SUBMITTER: Payne JM 

PROVIDER: S-EPMC6917317 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature