Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00843989%3A_____%2F23%3AE0110166" target="_blank" >RIV/00843989:_____/23:E0110166 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/23:10456269 RIV/00064203:_____/23:10456269
Result on the web
<a href="https://bmcnephrol.biomedcentral.com/articles/10.1186/s12882-023-03072-x" target="_blank" >https://bmcnephrol.biomedcentral.com/articles/10.1186/s12882-023-03072-x</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1186/s12882-023-03072-x" target="_blank" >10.1186/s12882-023-03072-x</a>
Alternative languages
Result language
angličtina
Original language name
Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease
Original language description
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 < 12 weeks in Study 204 and 28 days to < 18 years in Study 307. Subjects in both studies must have a clinical diagnosis of ARPKD, and those in Study 204 must additionally have signs indicative of risk of rapid progression to KRT, namely, all of: nephromegaly, multiple kidney cysts or increased kidney echogenicity suggesting microcysts, and oligohydramnios or anhydramnios. Target enrollment is 20 subjects for Study 204 and ? 10 subjects for Study 307. Results: Follow-up is 24 months in Study 204 (with optional additional treatment up to 36 months) and 18 months in Study 307. Outcomes include safety, tolerability, change in kidney function, and percentage of subjects requiring KRT relative to historical data. Regular safety assessments monitor for possible adverse effects of treatment on parameters such as liver function, kidney function, fluid balance, electrolyte levels, and growth trajectory, with increased frequency of monitoring following tolvaptan initiation or dose escalation. Conclusions: These trials will provide data on tolvaptan safety and efficacy in a population without disease-specific treatment options. Trial registration: Study 204: EudraCT 2020-005991-36; Study 307: EudraCT 2020-005992-10.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30209 - Paediatrics
Result continuities
Project
—
Continuities
N - Vyzkumna aktivita podporovana z neverejnych zdroju
Others
Publication year
2023
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
BMC Nephrology
ISSN
1471-2369
e-ISSN
1471-2369
Volume of the periodical
24
Issue of the periodical within the volume
article 33
Country of publishing house
GB - UNITED KINGDOM
Number of pages
11
Pages from-to
1-11
UT code for WoS article
000930554100001
EID of the result in the Scopus database
2-s2.0-85147912739