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)

Sponsor ID
156-12-204
Clinicaltrials.gov ID
NCT04786574
EudraCT ID
N/A
EUCTIS ID
N/A
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information
N/A
Terminated/Withdrawn
Sponsor ID
156-12-204
Trial Details
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Age
4 - 12 Weeks
Therapeutic Area
  • Kidney Disease
    • Sex
    Female & Male
    Trial Dates
    Jul 2022 - Dec 2025
    Healthy Volunteers
    No
    • 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.
    • 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

    Study 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)

    Additional Study Details

    Phase
    Phase 3
    Study Drug
    Drug: Tolvaptan (OPC-41061)
    Type
    Interventional
    Masking
    None (Open Label)
    Estimated Enrollment
    N/A
    Additional Study Details

    Inclusion and 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:

    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
    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

    Locations

    No locations found.

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