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Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease

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 trial...

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Main Authors: Mekahli, Djalila (Author) , Liebau, Max C. (Author) , Cadnapaphornchai, Melissa A. (Author) , Goldstein, Stuart L. (Author) , Greenbaum, Larry (Author) , Litwin, Mieczysław (Author) , Seeman, Tomas (Author) , Schaefer, Franz (Author) , Guay-Woodford, Lisa M. (Author)
Format: Article (Journal)
Language:English
Published: 13 February 2023
In: BMC nephrology
Year: 2023, Volume: 24, Pages: 1-11
ISSN:1471-2369
DOI:10.1186/s12882-023-03072-x
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s12882-023-03072-x
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Author Notes:Djalila Mekahli, Max C. Liebau, Melissa A. Cadnapaphornchai, Stuart L. Goldstein, Larry A. Greenbaum, Mieczyslaw Litwin, Tomas Seeman, Franz Schaefer and Lisa M. Guay-Woodford
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Summary: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.
Item Description:Gesehen am 19.04.2023
Physical Description:Online Resource
ISSN:1471-2369
DOI:10.1186/s12882-023-03072-x

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