Statistical Analyses
SECONDARY
A sample size of ≥60 participants aged 12–17 years was planned. It was expected that approximately 40 participants aged 4–11 years would also enroll for an anticipated total enrollment of 100 participants. As data were to be summarized descriptively, no formal power calculations to determine sample size were undertaken. All outcomes, including primary and secondary outcomes, were evaluated using observed values only (no missing data imputation). Missing data were to be mitigated by encouraging participants who withdrew from treatment to return for their remaining visits.The full analysis set, which was a modified intent-to-treat population, was used for the efficacy analyses. It consisted of all participants who were randomized to a treatment group, received ≥1 dose of the trial drug, and had both a phase A baseline and ≥1 postbaseline efficacy evaluation. The safety population consisted of all participants who received ≥1 dose of the trial drug. As the study was designed without formal efficacy testing, statistical comparisons between treatment groups (described in the 