Clinical Trial of Tolvaptan in Pediatric ADPKD: Two-Year, Open-Label Extension

Introduction

Tolvaptan is indicated for adults with autosomal dominant polycystic kidney disease (ADPKD) at increased risk of rapid progression. Pharmacodynamic activity and acceptable safety and tolerability were demonstrated in a 1-year, randomized, placebo-controlled trial in children and adolescents with ADPKD (Phase A of a 2-part, phase-3b study [NCT02964273]). Here, the 2-year, open-label extension (Phase B) is reported.