Dose escalations in phase I studies: Feasibility of interpreting blinded pharmacodynamic data.

ABSTRACT:

Aims

During phase I study conduct, blinded data are reviewed to predict the safety of increasing the dose level. The aim of the present study was to describe the probability that effects are observed in blinded evaluations of data in a simulated phase I study design.

Methods

An application was created to simulate blinded pharmacological response curves over time for 6 common safety/efficacy measurements in phase I studies for 1 or 2 cohorts (6 active, 2 placebo per cohort). Effect sizes between 0 and 3 between-measurement standard deviations (SDs) were simulated. Each set of simulated graphs contained the individual response and mean ± SD over time. Reviewers (n = 34) reviewed a median of 100 simulated datasets and indicated whether an effect was present.

Results

Incr

SUBMITTER: Hassing GJ 

PROVIDER: S-EPMC9805203 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature