Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Sponsor ID
156-12-298
Clinicaltrials.gov ID
NCT02964273
EudraCT ID
2016-000187-42
EUCTIS ID
N/A
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information
N/A
Study Complete
Sponsor ID
156-12-298
Trial Details
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Age
4 - 17 Years
Therapeutic Area
  • Kidney Disease
    • Sex
    Female & Male
    Trial Dates
    Sep 2016 - Nov 2021
    Healthy Volunteers
    No
    • Male and female participants aged 4 to 17 years (inclusive) with a diagnosis of ADPKD as defined by the presence of family history and/or genetic criteria AND who have at least 10 renal cysts, each of which measure at least 0.5 cm, confirmed upon magnetic resonance imaging (MRI) inspection; participants under the age of 12 years must have at least 4 cysts that are at least 1 cm in size, confirmed by ultrasound.
    • Weight ≥20 kg.
    • Participants with estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m\^2 within 31 days prior to randomization (using the Schwartz formula, eGFR = 0.413 × height \[cm\]/serum creatinine milligrams per deciliter \[mg/dL\]).
    • Independent in toileting.
    • Ability to swallow a tablet.
    • Liver function tests including aspartate aminotransferase (AST), alanine aminotransferase (ALT) \> 1.5 × the upper limit of normal (ULN).
    • Nocturnal enuresis.
    • Need for chronic diuretic use.
    • Participants with advanced diabetes (e.g., glycosylated hemoglobin \>7.5, and/or glycosuria by dipstick, significant proteinuria, retinopathy), evidence of additional significant renal disease(s) (i.e., currently active glomerular nephritides), renal cancer, single kidney, or recent (within 6 months of screening) renal surgery or acute kidney injury.
    • Participants having disorders in thirst recognition or inability to access fluids.
    • Participants with critical electrolyte imbalances, as determined by the investigator.
    • Participants with, or at risk of, significant hypovolemia as determined by investigator.
    • Participants with clinically significant anemia, as determined by investigator.
    • Participants 12 years of age and older having contraindications to, or interference with MRI assessments (e.g., ferro-magnetic prostheses, aneurysm clips, severe claustrophobia).
    • Participants with a history of taking a vasopressin agonist/antagonist.
    • Participants taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (i.e., marketed) therapies for the purpose of affecting polycystic kidney disease (PKD) cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (i.e., octreotide, sandostatin).
    • Participants who have had cyst reduction surgery within 6 weeks of the screening visit.

    Study Summary

    The primary objective of the study is to assess the long term safety of treatment with tolvaptan in children and adolescents with autosomal dominant polycystic kidney disease (ADPKD). The secondary objective is to assess the pharmacodynamics, pharmacokinetics, and efficacy of tolvaptan in the same participant population.

    Additional Study Details

    Phase
    Phase 3
    Study Drug
    Drug: Tolvaptan
    Type
    Interventional
    Masking
    Triple
    Estimated Enrollment
    91
    Additional Study Details

    Inclusion and Exclusion Criteria

    Inclusion Criteria

    • * Male and female participants aged 4 to 17 years (inclusive) with a diagnosis of ADPKD as defined by the presence of family history and/or genetic criteria AND who have at least 10 renal cysts, each of which measure at least 0.5 cm, confirmed upon magnetic resonance imaging (MRI) inspection; participants under the age of 12 years must have at least 4 cysts that are at least 1 cm in size, confirmed by ultrasound.
    • * Weight ≥20 kg.

    Exclusion Criteria

    • * Liver function tests including aspartate aminotransferase (AST), alanine aminotransferase (ALT) \> 1.5 × the upper limit of normal (ULN).
    • * Nocturnal enuresis.
    Inclusion and Exclusion Criteria
    Inclusion Criteria:
    • Male and female participants aged 4 to 17 years (inclusive) with a diagnosis of ADPKD as defined by the presence of family history and/or genetic criteria AND who have at least 10 renal cysts, each of which measure at least 0.5 cm, confirmed upon magnetic resonance imaging (MRI) inspection; participants under the age of 12 years must have at least 4 cysts that are at least 1 cm in size, confirmed by ultrasound.
    • Weight ≥20 kg.
    • Participants with estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m\^2 within 31 days prior to randomization (using the Schwartz formula, eGFR = 0.413 × height \[cm\]/serum creatinine milligrams per deciliter \[mg/dL\]).
    • Independent in toileting.
    • Ability to swallow a tablet.
    Exclusion Criteria:
    • Liver function tests including aspartate aminotransferase (AST), alanine aminotransferase (ALT) \> 1.5 × the upper limit of normal (ULN).
    • Nocturnal enuresis.
    • Need for chronic diuretic use.
    • Participants with advanced diabetes (e.g., glycosylated hemoglobin \>7.5, and/or glycosuria by dipstick, significant proteinuria, retinopathy), evidence of additional significant renal disease(s) (i.e., currently active glomerular nephritides), renal cancer, single kidney, or recent (within 6 months of screening) renal surgery or acute kidney injury.
    • Participants having disorders in thirst recognition or inability to access fluids.
    • Participants with critical electrolyte imbalances, as determined by the investigator.
    • Participants with, or at risk of, significant hypovolemia as determined by investigator.
    • Participants with clinically significant anemia, as determined by investigator.
    • Participants 12 years of age and older having contraindications to, or interference with MRI assessments (e.g., ferro-magnetic prostheses, aneurysm clips, severe claustrophobia).
    • Participants with a history of taking a vasopressin agonist/antagonist.
    • Participants taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (i.e., marketed) therapies for the purpose of affecting polycystic kidney disease (PKD) cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (i.e., octreotide, sandostatin).
    • Participants who have had cyst reduction surgery within 6 weeks of the screening visit.

    Study Documents

    Study Protocol
    Available Languages: English
    DOWNLOAD DOCUMENT(S)
    Statistical Analysis Plan
    Available Languages: English
    DOWNLOAD DOCUMENT(S)

    Locations

    Location
    Status
    Location
    Ghent, Oost-Vlaanderen, Belgium, 9000
    Status
    N/A
    Location
    Leuven, Vlaams Brabant, Belgium, 3000
    Status
    N/A
    Location
    Brussels, Belgium, 1020
    Status
    N/A
    Location
    Brussels, Belgium, 1200
    Status
    N/A
    Location
    Montegnée, Belgium, 4420
    Status
    N/A
    Location
    Cologne, Germany, 50937
    Status
    N/A
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