Historic Clinical Trial External Control Arm Provides Actionable GEN-1 Efficacy Estimate Before a Randomized Trial.

Xiang Yin Ruthanna C Davi Elizabeth B LamontPremal H ThakerWilliam H BradleyCharles A LeathKathleen M MooreKhursheed AnwerLauren MussoNicholas Borys

Published in: JCO clinical cancer informatics (2023)

Comparing results of a single-arm early-phase trial to those of a rigorously matched HCT ECA yielded insights regarding comparative efficacy prior to a randomized controlled trial. The effect size estimate itself informed both the decision to continue development and the randomized phase II trial (ClinicalTrials.gov identifier: NCT03393884) sample size. The work illustrates the potential of HCT data to inform drug development.

Keyphrases

open label
clinical trial
double blind
phase ii
placebo controlled
phase iii
cell cycle arrest
electronic health record
study protocol
randomized controlled trial
signaling pathway
risk assessment
data analysis