Statistical analysis
pruritus
SECONDARY
A sample size of 80 patients per treatment group, or 240 patients total, was estimated to provide (1) >90% power to detect a difference in the composite end point response rate (based on estimated response rates of <15%Efficacy was assessed in the modified intent-to-treat (mITT) population, which included all randomized patients who received ≥ 1 dose of study drug, and was analyzed based on randomized treatment. Efficacy analyses were conducted on the (mITT) population excluding patients who discontinued treatment before the month 3 assessment due to study closure. Patients who discontinued treatment before an assessment time point due to reasons other than study closure and did not have an assessment at the specified time point were classified as nonresponders. Safety was assessed during the study in all randomized patients who received ≥1 dose of study drug and was analyzed based on treatment received.Statistical comparisons of efficacy end points included the seladelpar 10- and 5-mg groups versus the placebo group and the seladelpar 10-mg group versus the 5-mg group for the primary and key secondary end points and mean change from baseline in ALP. Statistical tests were conducted using 2-sided tests at a 0.05 level of significance. The primary end point and ALP normalization response rate were analyzed using the Cochran-Mantel-Haenszel test adjusted for randomization stratification variables. Baseline pruritus NRS was defined as the mean of daily recorded scores during the run-in period. Assessment of pruritus NRS change from baseline compared the weekly averaged pruritus NRS of patients with a baseline score ≥4 using an analysis of covariance (ANCOVA) model. Least squares mean percent change from baseline for serum biochemistries was estimated using an ANCOVA model with percent change from baseline as the dependent variable, treatment group and randomization stratification factors as fixed effects, and baseline as a covariate.