A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
• Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
• Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
‣ Nephromegaly (\> 2 standard deviations from age-appropriate standard via ultrasound)
⁃ Multiple renal cysts
⁃ History of oligohydramnios or anhydramnios
• Ability for parent or guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial.