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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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • C.S. Mott Children's Hospital

    RECRUITING

    Ann Arbor, Michigan, 48109-5000, United States

  • Children's Hospital - New Orleans

    WITHDRAWN

    New Orleans, Louisiana, 70118, United States

  • Children's Hospital of Pittsburgh of UPMC

    WITHDRAWN

    Pittsburgh, Pennsylvania, 15213, United States

  • Children's National Medical Center

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Emory University Hospital

    WITHDRAWN

    Atlanta, Georgia, 30322, United States

  • Great Ormond Street Hospital for Children NHS Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Universitari Parc Tauli

    WITHDRAWN

    Sabadell, Barcelona, 08208, Spain

  • Hospital Universitari Vall D Hebron

    RECRUITING

    Barcelona, 8035, Spain

  • Hospital Universitario Virgen del Rocío Avenida Manuel Siurot

    WITHDRAWN

    Seville, 41013, Spain

  • Instytut "Pomnik - Centrum Zdrowia Dziecka"

    WITHDRAWN

    Warsaw, Masovian Voivodeship, 04-730, Poland

  • Johns Hopkins Pediatric Specialty Clinic

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • Mayo Clinic - Rochester

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology

    WITHDRAWN

    Chicago, Illinois, 60611, United States

  • Primary Children's Hospital

    WITHDRAWN

    Salt Lake City, Utah, 84113, United States

  • Riley Hospital for Children

    WITHDRAWN

    Indianapolis, Indiana, 46202-5119, United States

  • UZ Leuven

    RECRUITING

    Leuven, Vlaams Brabant, 3000, Belgium

  • Universitair Ziekenhuis Gent

    RECRUITING

    Ghent, Oost-Vlaanderen, 9000, Belgium

  • Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)

    RECRUITING

    Esplugues de Llobregat, Barcelona, 8950, Spain

  • Universitätsklinikum Köln

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Université Catholique De Louvain And Cliniques St Luc

    RECRUITING

    Brussels, Brussels Capital, 1200, Belgium

  • Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa

    WITHDRAWN

    Bialystok, 15-274, Poland

Conditions

Explore the condition pages connected to this study.