A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Study Identifier:
156-12-204
ClinicalTrials.gov Identifier:
NCT04786574
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information:
N/A
Terminated/Withdrawn
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Study Details

Medical Condition
  • Kidney Disease
Study Drug
  • Drug: Tolvaptan (OPC-41061)
Date
Jul 2022 - Dec 2025
Phase 1/2
Phase 2/3
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 4 - 12 Weeks
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
  • 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
  • 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
  • Premature birth (≤ 32 weeks gestational 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
  • Parents with renal cystic disease
  • Need for chronic diuretic use
  • Cannot be monitored for fluid balance
  • Has or at risk of having sodium and potassium electrolyte imbalances
  • Has or at risk of having significant hypovolemia as determined by investigator
  • Clinically significant anemia, as determined by investigator
  • 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
  • Having an infection including viral that would require therapy disruptive to IMP dosing
  • Platelet count \<50,000 µL
  • Significant Portal Hypertension
  • Bladder dysfunction or difficulty voiding
  • Taking vasopressin agonist
  • Having concomitant illness or taking medications that are likely to confound endpoint assessments.
  • History of cholangitis
  • Received or scheduled to receive a liver transplant

Protocol Summary

The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)

Study Locations

No locations found.
© 2025 Otsuka America Pharmaceutical,
Inc All rights reserved.

July 2025 01US25EUC0279

Requirements information
Inclusion criteria
  • Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
  • 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
  • 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
  • Premature birth (≤ 32 weeks gestational 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
  • Parents with renal cystic disease
  • Need for chronic diuretic use
  • Cannot be monitored for fluid balance
  • Has or at risk of having sodium and potassium electrolyte imbalances
  • Has or at risk of having significant hypovolemia as determined by investigator
  • Clinically significant anemia, as determined by investigator
  • 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
  • Having an infection including viral that would require therapy disruptive to IMP dosing
  • Platelet count \<50,000 µL
  • Significant Portal Hypertension
  • Bladder dysfunction or difficulty voiding
  • Taking vasopressin agonist
  • Having concomitant illness or taking medications that are likely to confound endpoint assessments.
  • History of cholangitis
  • Received or scheduled to receive a liver transplant

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