Calculation of test-retest variability in phase I/IIa clinical trials for Inherited Retinal Degenerations.

ABSTRACT: Background: Several novel treatments of inherited retinal degenerations have undergone phase I/IIa clinical trials with limited sample size, yet investigators must still determine if toxicity or an efficacy signal occurred or if the change was due to test-retest variability (TRV) of the measurement tool.Materials and Methods: Synthetic datasets were used to compare three types of TRV estimators under different sample sizes, mean drift, skewness, and number of baseline measurements.Results: Mixed effects models underestimated the standard deviation of measurement error (SDEM); the unbiased change score estimator method (UBS) was more accurate. The fixed effect model had less bias and smaller standard deviation than UBS if >2 baseline measurements. The change score estim

SUBMITTER: Abou-Hanna JJ 

PROVIDER: S-EPMC8507608 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature