Tolvaptan use in children and adolescents with autosomal dominant polycystic kidney disease: rationale and design of a two-part, randomized, double-blind, placebo-controlled trial

Conclusion: This will be the first clinical study to evaluate tolvaptan in pediatric ADPKD.What is Known:• Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder causing the development of cysts that impede kidney function over time and eventually induce renal failure• There are few data on the effects of tolvaptan, the only treatment approved for adults to slow disease progression, in pediatric ADPKD patients with early-stage diseaseWhat is New:• A phase 3, placebo-controlled study is evaluating tolvaptan over 3 years in children and adolescents with ADPKD• This study is designed to account for challenges of tolvaptan dosing and outcome assessment specific to the pediatric population
Source: European Journal of Pediatrics - Category: Pediatrics Source Type: research