New hope for kids with rare kidney disease: drug trial launches
NCT ID NCT04782258
First seen Nov 01, 2025 · Last updated May 10, 2026 · Updated 21 times
Summary
This study tests the safety of a drug called tolvaptan in children (28 days to under 18 years) with autosomal recessive polycystic kidney disease (ARPKD), a rare genetic condition that causes kidney cysts. About 20 participants will take the drug for 18 months to see if it helps slow kidney decline. The goal is to manage the disease, not cure it.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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C.S. Mott Children's Hospital
RECRUITINGAnn Arbor, Michigan, 48109-5000, United States
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Children's Hospital - New Orleans
WITHDRAWNNew Orleans, Louisiana, 70118, United States
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Children's Hospital of Pittsburgh of UPMC
WITHDRAWNPittsburgh, Pennsylvania, 15213, United States
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Children's National Medical Center
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-3039, United States
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Cleveland Clinic
RECRUITINGCleveland, Ohio, 44195, United States
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Emory University Hospital
WITHDRAWNAtlanta, Georgia, 30322, United States
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Great Ormond Street Hospital for Children NHS Trust
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Universitari Parc Tauli
WITHDRAWNSabadell, Barcelona, 08208, Spain
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Hospital Universitari Vall D Hebron
RECRUITINGBarcelona, 8035, Spain
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Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
WITHDRAWNSeville, 41013, Spain
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Instytut "Pomnik - Centrum Zdrowia Dziecka"
WITHDRAWNWarsaw, Masovian Voivodeship, 04-730, Poland
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Johns Hopkins Pediatric Specialty Clinic
RECRUITINGBaltimore, Maryland, 21287, United States
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Mayo Clinic - Rochester
RECRUITINGRochester, Minnesota, 55905, United States
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Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
WITHDRAWNChicago, Illinois, 60611, United States
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Primary Children's Hospital
WITHDRAWNSalt Lake City, Utah, 84113, United States
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Riley Hospital for Children
WITHDRAWNIndianapolis, Indiana, 46202-5119, United States
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UZ Leuven
RECRUITINGLeuven, Vlaams Brabant, 3000, Belgium
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Universitair Ziekenhuis Gent
RECRUITINGGhent, Oost-Vlaanderen, 9000, Belgium
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Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
RECRUITINGEsplugues de Llobregat, Barcelona, 8950, Spain
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Universitätsklinikum Köln
RECRUITINGCologne, North Rhine-Westphalia, 50937, Germany
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Université Catholique De Louvain And Cliniques St Luc
RECRUITINGBrussels, Brussels Capital, 1200, Belgium
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Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
WITHDRAWNBialystok, 15-274, Poland
Conditions
Explore the condition pages connected to this study.