A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)
NCT ID: NCT04782258
Last Updated: 2026-01-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
20 participants
INTERVENTIONAL
2023-01-23
2028-08-14
Brief Summary
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Detailed Description
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Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 4), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).
Participants in this study will be assigned to tolvaptan and followed for 18 months over the course of the study.
The overall trial duration is expected to be approximately 3.5 years.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Tolvaptan Suspension
Tolvaptan suspension will be administered orally or via feeding/nasogastric tube at doses of 0.15 mg/kg once daily in the AM, 0.30 mg/kg once daily in the AM, 0.5 mg/kg once daily in the AM, 0.75 mg/kg split dose (0.5 mg/kg AM and 0.25 mg/kg 8 hours later), and 1 mg/kg split dose (0.67 mg/kg AM and 0.33 mg/kg 8 hours later) based on age. Treatment duration is 18 months.
Tolvaptan Suspension
Syrup
Tolvaptan Tablets
Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets. Treatment duration is 18 months.
Tolvaptan Tablets
Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.
Interventions
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Tolvaptan Suspension
Syrup
Tolvaptan Tablets
Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.
Eligibility Criteria
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Inclusion Criteria
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
2. Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
4. Abnormal liver function tests including ALT and AST, \> 1.2 × ULN (upper limit of normal).
5. Has splenomegaly or portal hypertension (HTN).
6. Parents with renal cystic disease.
7. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
8. Cannot be monitored for fluid balance.
9. Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
10. Has or at risk of having significant hypovolemia as determined by investigator.
11. Clinically significant anemia, as determined by investigator.
12. Platelets \< 50000 µL.
13. Severe systolic dysfunction defined as ejection fraction \< 14%.
14. Serum sodium levels \< 130 mmol/L or \>145 mmol/L.
15. Taking any other experimental medications.
16. Require ventilator support.
17. Taking medications known to induce CYP3A4 (CYP = Cytochrome P).
18. Having an infection including viral that would require therapy disruptive to IMP dosing.
19. Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.
20. Subjects with a history of substance abuse (within the last 6 months).
21. Subjects who have bladder dysfunction and/or difficulty voiding.
22. Subjects taking a vasopressin agonist (eg, desmopressin).
23. Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy.
24. 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).
25. Received or are scheduled to receive a liver transplant.
26. History of cholangitis within the last 6 months.
27. Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).
28 Days
18 Years
ALL
No
Sponsors
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Otsuka Pharmaceutical Development & Commercialization, Inc.
INDUSTRY
Responsible Party
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Locations
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Children's National Medical Center
Washington D.C., District of Columbia, United States
Emory University Hospital
Atlanta, Georgia, United States
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
Children's Hospital - New Orleans
New Orleans, Louisiana, United States
Johns Hopkins Pediatric Specialty Clinic
Baltimore, Maryland, United States
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Université Catholique De Louvain And Cliniques St Luc
Brussels, Brussels Capital, Belgium
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, Belgium
UZ Leuven
Leuven, Vlaams Brabant, Belgium
Universitätsklinikum Köln
Cologne, North Rhine-Westphalia, Germany
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, Masovian Voivodeship, Poland
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
Bialystok, , Poland
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitari Parc Tauli
Sabadell, Barcelona, Spain
Hospital Universitari Vall D Hebron
Barcelona, , Spain
Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
Seville, , Spain
Great Ormond Street Hospital for Children NHS Trust
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2020-005992-10
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
156-201-00307
Identifier Type: -
Identifier Source: org_study_id
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