A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Sponsor ID
156-201-00307
Clinicaltrials.gov ID
NCT04782258
EudraCT ID
2020-005992-10
EUCTIS ID
N/A
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information
844-687-8522 or [email protected]
Recruiting
Sponsor ID
156-201-00307
Trial Details
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Age
28 Days - 18 Years
Therapeutic Area
  • Kidney Disease
    • Sex
    Female & Male
    Trial Dates
    Jan 2023 - Jul 2028
    Healthy Volunteers
    No
    • 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.
    • 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.
    • 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 (Investigational Medicinal Product) 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).
    • Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP:
    • Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide
    • Intrauterine device
    • Hormone-based contraceptives which are associated with inhibition of ovulation.

    Study Summary

    To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD

    Additional Study Details

    Phase
    Phase 3
    Study Drug
    Drug: Tolvaptan Suspension
    Type
    Interventional
    Masking
    None (Open Label)
    Estimated Enrollment
    20
    Additional Study Details

    Inclusion and 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.
    Inclusion and Exclusion Criteria
    Inclusion Criteria:
    • 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.
    • 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 (Investigational Medicinal Product) 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).
    • Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP:
    • Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide
    • Intrauterine device
    • Hormone-based contraceptives which are associated with inhibition of ovulation.

    Locations

    Location
    Status
    Location
    Children's National Medical Center
    Washington D.C., District of Columbia, United States, 20010
    Status
    Recruiting
    Location
    Emory University Hospital
    Atlanta, Georgia, United States, 30322
    Status
    Withdrawn
    Location
    Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
    Chicago, Illinois, United States, 60611
    Status
    Withdrawn
    Location
    Riley Hospital for Children
    Indianapolis, Indiana, United States, 46202-5119
    Status
    Withdrawn
    Location
    Children's Hospital - New Orleans
    New Orleans, Louisiana, United States, 70118
    Status
    Withdrawn
    Location
    Johns Hopkins Pediatric Specialty Clinic
    Baltimore, Maryland, United States, 21287
    Status
    Recruiting
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    January 2026 01US25EUC0489

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