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Hope for kids with rare kidney disease: drug trial aims to slow damage

NCT ID NCT04782258

Recruiting now Disease control Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tests the safety of tolvaptan, a drug that helps the body remove extra water, in 20 children aged 28 days to 18 years with autosomal recessive polycystic kidney disease (ARPKD). The goal is to see if the drug can slow kidney function decline and reduce the need for dialysis or transplant. The study is open-label, meaning everyone gets the drug, and will monitor side effects closely.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Tolvaptan (a drug that helps the kidneys remove excess water)

What this could lead to

If it works, this could provide a treatment option to slow kidney damage and delay the need for dialysis or transplant in children with ARPKD.

What could go wrong

This is a small, early-phase trial with only 20 children, so results may not apply to all. Tolvaptan can cause liver injury and other side effects, and it is not a cure.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

autosomal recessive polycystic kidney disease oligohydramnios polycystic kidney disease Polycystic Kidney, Autosomal Recessive

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AUTOSOMAL RECESSIVE POLYCYSTIC KIDNEY (ARPKD)

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    WITHDRAWN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    WITHDRAWN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