Kidney international reports
Clinical Trial of Tolvaptan in Pediatric ADPKD: Two-Year, Open-Label Extension
By Mekahli D, Guay-Woodford LM, Cadnapaphornchai MA and 10 others.
Abstract
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.
Methods
Phase A participants could enter Phase B if they had not experienced adverse events requiring discontinuation. All Phase B participants received tolvaptan, titrated based on body weight and tolerability. Outcomes included safety, tolerability, kidney function, height-adjusted total kidney volume (htTKV), and health-related quality of life (HRQoL).