End Points
early-stage disease
Given that treatment effects on TKV or kidney function are difficult to measure in a pediatric population with early-stage disease, pharmacodynamic parameters were considered the most appropriate coprimary end points: change from baseline in spot urine osmolality and spot urine specific gravity at week 1 in all participants. Spot urine samples were collected before the morning dose and before eating or drinking anything for breakfast from the urine void (second-morning void) taken after the first morning's void and ideally collected as a mid-stream, clean catch. The samples were analyzed as described in the 