Critical design considerations for time-to-event endpoints in amyotrophic lateral sclerosis clinical trials.

Ruben P A van EijkStavros NikolakopoulosKit C B RoesBas M MiddelkoopToby A FergusonPamela J ShawP Nigel LeighAmmar Al-ChalabiMarinus J C EijkemansLeonard H van den Berg

Published in: Journal of neurology, neurosurgery, and psychiatry (2019)

Implementing distributional knowledge and incorporating accrual at the design stage could achieve large gains in the efficiency of ALS clinical trials with time-to-event endpoints. We provide an open-source platform that helps investigators to make more accurate sample size calculations and optimise the use of their available resources.

Keyphrases

clinical trial
healthcare
phase ii
high throughput
density functional theory
molecular dynamics
molecular dynamics simulations
open label
study protocol
quality improvement
amyotrophic lateral sclerosis
single cell