Statistical plan and analysis
SECONDARY
Full analysis set and intent-to-treat population (FAS/ITTP) was defined as all patients who were randomly allocated to a study arm. The primary efficacy analysis was based on the FAS/ITT population. The primary endpoint was analyzed by Wilcoxon test stratified by its baseline values with a two-sided α-level of 5%. The secondary endpoints were tested after a significant test of the primary endpoint (Quantitative secondary study parameters were described based on their mean, standard deviation (SD), median, interquartile range (IQR), minima, and maxima and portrayed by Kaplan–Meier plots and compared with the log rank test. Qualitative secondary study parameters were analyzed by means of absolute and relative frequencies: Chi-square tests were carried out in order to compare the active treatments to SOC. With a two-sided significance level of α = 0.05 and a power of 1 − β = 0.80, a sample size of 76 (2 × 38) was calculated for a fixed sample size design and an advantage of at least 1 point on the WHO Ordinal Scale (SD 1.5 points) (The independent clinical trial statistician of CRM Biometrics GmbH, the first author (PU), and last author (MO) analyzed the data and had access to all data. All authors had access to primary clinical trial data only related to their institution. Study participants, treating physicians, nurses, investigators, and DSMB members were not blinded.