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

Study Identifier:
156-12-298
ClinicalTrials.gov Identifier:
NCT02964273
EudraCT Identifier:
2016-000187-42
EU CT ID:
N/A
Sponsor:
Otsuka Pharmaceutical Development & Commercialization, Inc.
Study Contact Information:
N/A
Study Complete
Subscribe to Updates

Results Availables

Study Protocol
Available Language: English
Download document(s)
Statistical Analysis Plan
Available Language: English
Download document(s)

Study Details

Medical Condition
  • Kidney Disease
Study Drug
  • Drug: Tolvaptan
  • Drug: Tolvaptan Matching-placebo
Date
Sep 2016 - Nov 2021
Phase 1/2
Phase 2/3
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 4 - 17 Years
Requirements Information
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.

Protocol 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.

Study 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
Go to page
© 2025 Otsuka America Pharmaceutical,
Inc All rights reserved.

July 2025 01US25EUC0279

Requirements information
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.

We defy limitation,

so that others can too.

Follow Us
OAPI and OPDC Locations

508 Carnegie Center Dr,

Princeton, NJ 08540, United States

Phone 609-524-6788

2440 Research Blvd,

Rockville, MD 20850, United States

Phone 301-424-9055

3956 Point Eden Way

Hayward, CA 94545, United States

Phone 800-562-3974