학술논문
Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease
Document Type
article
Author
Source
BMC Nephrology, Vol 24, Iss 1, Pp 1-11 (2023)
Subject
Language
English
ISSN
1471-2369
Abstract
Abstract Purpose Autosomal recessive polycystic kidney disease (ARPKD) is a hereditary condition characterized by massive kidney enlargement and developmental liver defects. Potential consequences during childhood include the need for kidney replacement therapy (KRT). We report the design of 2 ongoing clinical trials (Study 204, Study 307) to evaluate safety, tolerability, and efficacy of tolvaptan in children with ARPKD. Methods Both trials are of multinational, multicenter, open-label design. Age range at enrollment is 28 days to