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Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD

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 subject population.

Intervention:

Drug: Tolvaptan
Drug: Matching Placebo

Participation criteria:

Inclusion cirteria:

  • Male and female subjects 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; subjects under the age of 12 years must have at least 4 cysts that are at least 1 cm in size, confirmed by ultrasound.
  • Male and female subjects 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; subjects 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.
  • Subjects with estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m2 within 31 days prior to randomization (using the Schwartz formula, eGFR = 0.413 × height [cm]/serum creatinine mg/dL).
  • Independent in toileting.
  • Ability to swallow a tablet.

Exclusion criteria:

  • Liver function tests including AST (aspartate aminotransferase), ALT (alanine aminotransferase) > 1.5 × the upper limit of normal (ULN).
  • Nocturnal enuresis.
  • Need for chronic diuretic use.
  • Subjects with advanced diabetes (eg, glycosylated hemoglobin > 7.5, and/or glycosuria by dipstick, significant proteinuria, retinopathy), evidence of additional significant renal disease(s) (ie, currently active glomerular nephritides), renal cancer, single kidney, or recent (within 6 months of screening) renal surgery or acute kidney injury.
  • Subjects having disorders in thirst recognition or inability to access fluids.
  • Subjects with critical electrolyte imbalances, as determined by the investigator.
  • Subjects with, or at risk of, significant hypovolemia as determined by investigator.
  • Subjects with clinically significant anemia, as determined by investigator.
  • Subjects 12 years of age and older having contraindications to, or interference with MRI assessments (eg, ferro-magnetic prostheses, aneurysm clips, severe claustrophobia).
  • Subjects with a history of taking a vasopressin agonist/antagonist.
  • Subjects taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, 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 (ie, octreotide, sandostatin).
  • Subjects who have had cyst reduction surgery within 6 weeks of the screening visit.

Contact information:

Taylor Cook
919-397-5305
Taylor.Cook@INCResearch.com

Sarah van Leeuwen
+310203018569
Sarah.vanLeeuwen@INCResearch.com