The STAGED-PKD trial (Study To Assess Glucosylceramide synthase inhibitor Efficacy in ADPKD; NCT03523728) is an international, multicenter, randomized, double-blind, placebo-controlled study designed to characterize the efficacy, safety, tolerability, and pharmacokinetics of venglustat in patients with rapidly progressing ADPKD. Here, UGCG is linked to autosomal dominant polycystic kidney disease.