A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

August 4, 2026 updated by: Otsuka Pharmaceutical Development & Commercialization, Inc.

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study is a multinational, multicenter, open-label, non-randomized trial. The study consist of three periods: Screening Period, Treatment period and Follow-up period.

Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 4), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).

Participants in this study will be assigned to tolvaptan and followed for 24 months over the course of the study.

The overall trial duration is expected to be approximately 5 years.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
    • Brussels Capital
      • Brussels, Brussels Capital, Belgium, 1200
        • Recruiting
        • Université Catholique De Louvain And Cliniques St Luc
    • Oost-Vlaanderen
      • Ghent, Oost-Vlaanderen, Belgium, 9000
        • Recruiting
        • Universitair Ziekenhuis Gent
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • Recruiting
        • UZ Leuven
  • Germany
    • North Rhine-Westphalia
      • Cologne, North Rhine-Westphalia, Germany, 50937
        • Recruiting
        • Universitatsklinikum Koln
  • Poland
      • Bialystok, Poland, 15-274
        • Withdrawn
        • Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
    • Masovian Voivodeship
      • Warsaw, Masovian Voivodeship, Poland, 04-730
        • Withdrawn
        • Instytut "Pomnik - Centrum Zdrowia Dziecka"
  • Spain
      • Barcelona, Spain, 8035
        • Recruiting
        • Hospital Universitari Vall D Hebron
      • Seville, Spain, 41013
        • Withdrawn
        • Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spain, 8950
        • Recruiting
        • Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
      • Sabadell, Barcelona, Spain, 08208
        • Withdrawn
        • Hospital Universitari Parc Tauli
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital for Children NHS Trust
  • United States
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Children's National Medical Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Withdrawn
        • Emory University Hospital
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Withdrawn
        • Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5119
        • Withdrawn
        • Riley Hospital for Children
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Withdrawn
        • Children's Hospital - New Orleans
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Recruiting
        • Johns Hopkins Pediatric Specialty Clinic
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5000
        • Recruiting
        • C.S. Mott Children's Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic - Rochester
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Withdrawn
        • Children's Hospital of Pittsburgh of UPMC
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Withdrawn
        • Primary Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

4 weeks to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Tolvaptan Suspension
Tolvaptan suspension will be administered orally or via feeding/nasogastric tube at doses of 0.15 mg/kg once daily in the AM, 0.30 mg/kg once daily in the AM, 0.5 mg/kg once daily in the AM, 0.75 mg/kg split dose (0.5 mg/kg AM and 0.25 mg/kg 8 hours later), and 1 mg/kg split dose (0.67 mg/kg AM and 0.33 mg/kg 8 hours later) based on age. Treatment duration is 24 months.
Drug: Tolvaptan Suspension
Syrup
Experimental: Tolvaptan Tablets
Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets. Treatment duration is 24 months.
Drug: Tolvaptan Tablets
Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)
Time Frame: From baseline to post-treatment after 24 months or EoTx
Number of participants with TEAEs will be assessed from Baseline to post-treatment after 24 months or End of Treatment (EOTx). The EOTx visit applies to participants who discontinue IMP before Month 24. TEAEs are defined as Adverse Events (AEs) with an onset date on or after the start of Investigational Medicinal Product (IMP) treatment. TEAEs are also events continuous from baseline which worsened, became serious, were IMP related, or resulted in death, discontinuation, interruption, or reduction of IMP. An AE is defined as any untoward medical occurrence in a clinical trial subject administered an IMP and which does not necessarily have a causal relationship with this treatment.
From baseline to post-treatment after 24 months or EoTx

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
The amount of time between enrollment and 24 months that a subject requires renal replacement therapy (RRT).
Time Frame: From enrollment to 24 months
To evaluate the effect of tolvaptan on the need for RRT in pediatric participants with ARPKD.
From enrollment to 24 months
Annual rate of change of eGFR (by Schwartz formula) from baseline to post-treatment after 24 months
Time Frame: From Baseline to post-treatment after Month 24 or EOTx
Annual rate of change of eGFR from Baseline to post-treatment after Month 24 or EOTx is calculated using eGFR Schwartz formula = 0.413 × height [or length, centimeter (cm)] /serum creatinine milligram per deciliter (mg/dL). The EOTx visit applies to participants who discontinue IMP before Month 24.
From Baseline to post-treatment after Month 24 or EOTx
Change from baseline of eGFR (by Schwartz formula) while on treatment at Months 1, 6, 12, 18 and 24 or EOTx
Time Frame: At Months 1, 6, 12, 18, and 24 or EOTx
Change from baseline of eGFR is calculated using eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) while on treatment at Months 1, 6, 12, 18, and 24 or EoTx. The EOTx visit applies to participants who discontinue IMP before Month 24.
At Months 1, 6, 12, 18, and 24 or EOTx
The percentage of subjects that will receive renal replacement therapy (RRT) by 24 months
Time Frame: From Baseline to Month 24
Percentage of subjects who receive RRT from baseline through Month 24. RRT is defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or kidney transplantation.
From Baseline to Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Otsuka Pharmaceutical Development & Commercialization, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Olga Sergeyeva, MD, Olga.Sergeyeva@otsuka-us.com

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 23, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 31, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 14, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 19, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 3, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 4, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 7, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 156-201-00307
  • 2020-005992-10 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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