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)

  • End date
    Apr 27, 2026
  • participants needed
  • sponsor
    Otsuka Pharmaceutical Development & Commercialization, Inc.
Updated on 27 October 2022


The primary objective of this study is to evaluate the safety of tolvaptan in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)


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 18 months over the course of the study.

The overall trial duration is expected to be approximately 3.5 years.

Condition Autosomal Recessive Polycystic Kidney (ARPKD)
Treatment Tolvaptan Tablets, Tolvaptan Suspension
Clinical Study IdentifierNCT04782258
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.
Last Modified on27 October 2022


Yes No Not Sure

Inclusion Criteria

Male or female subjects between 28 days and less than 18 years of age, with 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; and a history of oligohydramnios or anhydramnios in utero
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

Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age
Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation
Evidence of syndromic conditions associated with renal cysts (other than ARPKD)
Abnormal liver function tests including ALT and AST, > 1.2 × ULN (upper limit of normal)
Has splenomegaly or portal hypertension (HTN)
Parents with renal cystic disease
Receiving chronic diuretic that could not be adjusted after tolvaptan initiation
Cannot be monitored for fluid balance
Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator
Has or at risk of having significant hypovolemia as determined by investigator
Clinically significant anemia, as determined by investigator
Platelets < 50000 µL
Severe systolic dysfunction defined as ejection fraction < 14%
Serum sodium levels < 130 mmol/L or >145 mmol/L
Taking any other experimental medications
Require ventilator support
Taking medications known to induce CYP3A4 (CYP = Cytochrome P)
Having an infection including viral that would require therapy disruptive to IMP dosing
Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP
Subjects with a history of substance abuse (within the last 6 months)
Subjects who have bladder dysfunction and/or difficulty voiding
Subjects taking a vasopressin agonist (eg, desmopressin)
Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy
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)
Received or are scheduled to receive a liver transplant
History of cholangitis within the last 6 months
Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia)
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