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 Aug 07, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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

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WITHDRAWN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WITHDRAWN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WITHDRAWN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