Statistical considerations
PD
Sample size determination followed practical, feasibility, and empirical considerations for a Phase 2 dose-finding study. The planned enrollment was 32 patients with 16 patients in each treatment group equally distributed by age (eight patients aged 1–6 years and eight aged >6–17 years).The safety population included all patients who received FCM; the PK population included all patients in the safety population who had at least one measurable concentration of FCM; the PD population included all patients in the safety population who had at least one PD assessment. Baseline was defined as Day 0; if data on Data 0 were missing or not captured, the screening value was used.No hypothesis testing was planned for this study; only descriptive, summary statistics were planned for assessment of dosing and safety. Statistical programming and analyses were performed using SAS v9.1.3 (SAS Institute Inc., Cary, NC).