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Using tolvaptan to delay dialysis in infants and 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 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Phase 3 Interventional Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT04786574

This study is testing if the medication tolvaptan can help delay the need for dialysis in infants and children with a kidney disease called ARPKD.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment20 (estimated)
Ages28 Days to 12 Weeks
SexAll
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc. Industry-sponsored
Locations18 sites (Washington, District of Columbia and 17 other locations)
Trial IDNCT04786574 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness of tolvaptan in delaying the need for renal replacement therapy in infants and children diagnosed with autosomal recessive polycystic kidney disease (ARPKD). Participants aged between 28 days and less than 12 weeks will receive tolvaptan for a duration of 24 months, with close monitoring of their kidney function and overall health. The study aims to build on previous findings in adults with a related condition, aiming to provide a new treatment option for pediatric patients with ARPKD.

Who should consider this trial

Good fit: Ideal candidates are infants and children aged 28 days to less than 12 weeks with a confirmed diagnosis of ARPKD.

Not a fit: Patients who are premature (≤ 32 weeks gestational age) or already require renal replacement therapy will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly delay the onset of dialysis in young patients with ARPKD, improving their quality of life.

How similar studies have performed: While this is the first trial of tolvaptan specifically in pediatric ARPKD, similar approaches in adults with related conditions have shown promising results.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
2. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:

   * Nephromegaly (\> 2 standard deviations from age appropriate standard via ultrasound)
   * Multiple renal cysts
   * History of oligohydramnios or anhydramnios
3. Ability for parent or 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.

Exclusion Criteria:

1. Premature birth (≤ 32 weeks gestational 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
5. Parents with renal cystic disease
6. Need for chronic diuretic use
7. Cannot be monitored for fluid balance
8. Has or at risk of having sodium and potassium electrolyte imbalances
9. Has or at risk of having significant hypovolemia as determined by investigator
10. Clinically significant anemia, as determined by investigator
11. Severe systolic dysfunction defined as ejection fraction \< 14%
12. Serum sodium levels \< 130 mmol/L or \>145 mmol/L
13. Taking any other experimental medications
14. Require ventilator support
15. Taking medications known to induce CYP3A4
16. Having an infection including viral that would require therapy disruptive to IMP dosing
17. Platelet count \<50,000 µL
18. Significant Portal Hypertension
19. Bladder dysfunction or difficulty voiding
20. Taking vasopressin agonist
21. Having concomitant illness or taking medications that are likely to confound endpoint assessments.
22. History of cholangitis
23. Received or scheduled to receive a liver transplant

Where this trial is running

Washington, District of Columbia and 17 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 Kidney DiseaseARPKDTolvaptanPolycystic Kidney DiseaseRenal CystsNephromegalyOligohydramniosAnhydramnios
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.