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Evaluating the safety of tolvaptan in children with ARPKD

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)

Phase 3 Interventional Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT04782258

This study is testing if the medication tolvaptan is safe for children with autosomal recessive polycystic kidney disease (ARPKD) over an 18-month period.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment20 (estimated)
Ages28 Days to 18 Years
SexAll
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc. Industry-sponsored
Locations23 sites (Washington D.C., District of Columbia and 22 other locations)
Trial IDNCT04782258 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety of tolvaptan in pediatric patients diagnosed with autosomal recessive polycystic kidney disease (ARPKD). It is a multinational, multicenter, open-label, non-randomized study that includes a screening period, a treatment period with tolvaptan, and a follow-up period. Participants will be monitored for 18 months to evaluate the drug's effects on kidney function and overall health. The trial is expected to last approximately 3.5 years.

Who should consider this trial

Good fit: Ideal candidates for this study are infants and children aged 28 days to less than 18 years with a confirmed diagnosis of ARPKD.

Not a fit: Patients who have undergone kidney transplantation or have severe liver dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option that may improve kidney function and quality of life for children with ARPKD.

How similar studies have performed: Previous studies have shown that tolvaptan can delay kidney function decline in adults with a related condition, suggesting potential for success in this pediatric population.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 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).

Where this trial is running

Washington D.C., District of Columbia and 22 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Autosomal Recessive Polycystic KidneyARPKDTOLVAPTANPolycystic Kidney DiseaseAutosomal Recessive Polycystic Kidney DiseaseAdolescentRenal CystsOligohydramnios
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.