Fig. 1

a Staggered approach and sampling strategy for collecting PK/PD and safety data in pediatric cohorts in this phase I study. PK/PD and safety data available for at least four subjects before starting next cohort; blue arrows indicate data monitoring committee agreement to progress to the next planned step of the phase I study, i.e. data collection for the next cohort. b Sampling windows per age group eq., equivalent, PD pharmacodynamics, PK pharmacokinetics.