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

NCT ID NCT04782258

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2023

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

28 days to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD. 2. Ability for parent/legal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent. Exclusion Criteria: 1. Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of age. 2. Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation. 3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD). 4. Abnormal liver function tests including ALT and AST, \> 1.2 × ULN (upper limit of normal). 5. Has splenomegaly or portal hypertension (HTN). 6. Parents with renal cystic disease. 7. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation. 8. Cannot be monitored for fluid balance. 9. Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator. 10. Has or at risk of having significant hypovolemia as determined by investigator. 11. Clinically significant anemia, as determined by investigator. 12. Platelets \< 50000 µL. 13. Severe systolic dysfunction defined as ejection fraction \< 14%. 14. Serum sodium levels \< 130 mmol/L or \>145 mmol/L. 15. Taking any other experimental medications. 16. Require ventilator support. 17. Taking medications known to induce CYP3A4 (CYP = Cytochrome P). 18. Having an infection including viral that would require therapy disruptive to IMP (Investigational Medicinal Product) dosing. 19. Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP. 20. Subjects with a history of substance abuse (within the last 6 months). 21. Subjects who have bladder dysfunction and/or difficulty voiding. 22. Subjects taking a vasopressin agonist (eg, desmopressin). 23. Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy. 24. Subjects taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin). 25. Received or are scheduled to receive a liver transplant. 26. History of cholangitis within the last 6 months. 27. Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia). 28. Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP: * Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide * Intrauterine device * Hormone-based contraceptives which are associated with inhibition of ovulation.

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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)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    13 sites in 5 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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