Everolimus: Phase III data

In a double-blind Phase III trial in 433 patients, everolimus significantly reduced increases in total kidney volume from baseline to 1 year (102 vs. 157 mL, p=0.02), but non-significantly reduced volume increases at 2 years (230 vs. 301 mL, p=0.06). Cyst volume increased by 76 mL for everolimus at 1 year vs. 98 mL for placebo (p=0.27), and by 181 and 215 mL, respectively, at 2 years (p=0.28). Parenchymal volume increased by 26 mL for everolimus at 1 year vs. 62 mL for placebo (p=0.003), and by 56 and 93 mL, respectively, at 2 years (p=0.11). Data were published in the New England Journal of Medicine. ...