🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Clinical Trials in the USA / NCT04782258
Recruiting Phase 3

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)

NCT04782258 · tracked via the Priya Life Science USA tracker
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Phase
Phase 3
Started
2023-01-23
Last updated
2026-08-07

Condition(s) studied

Autosomal Recessive Polycystic Kidney (ARPKD)

Investigational drug(s) / intervention(s)

Tolvaptan SuspensionTolvaptan Tablets

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.

Study summary

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

Eligibility

Sex
ALL
Min age
28 Days
Max age
18 Years
Healthy volunteers
No
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. 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 (Investigational Medicinal Product) 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. 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.

Primary outcome measure(s)

Trial sites (23)

FacilityCityRegionStatus
Children's National Medical Center Washington D.C. District of Columbia Recruiting
Emory University Hospital Atlanta Georgia Withdrawn
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology Chicago Illinois Withdrawn
Riley Hospital for Children Indianapolis Indiana Withdrawn
Children's Hospital - New Orleans New Orleans Louisiana Withdrawn
Johns Hopkins Pediatric Specialty Clinic Baltimore Maryland Recruiting
C.S. Mott Children's Hospital Ann Arbor Michigan Recruiting
Mayo Clinic - Rochester Rochester Minnesota Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Cleveland Clinic Cleveland Ohio Recruiting
Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania Withdrawn
Primary Children's Hospital Salt Lake City Utah Withdrawn
Université Catholique De Louvain And Cliniques St Luc Brussels Brussels Capital Recruiting
Universitair Ziekenhuis Gent Ghent Oost-Vlaanderen Recruiting
UZ Leuven Leuven Vlaams Brabant Recruiting
Universitätsklinikum Köln Cologne North Rhine-Westphalia Recruiting
Instytut "Pomnik - Centrum Zdrowia Dziecka" Warsaw Masovian Voivodeship Withdrawn
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa Bialystok Poland Withdrawn
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB) Esplugues de Llobregat Barcelona Recruiting
Hospital Universitari Parc Tauli Sabadell Barcelona Withdrawn
Hospital Universitari Vall D Hebron Barcelona Spain Recruiting
Hospital Universitario Virgen del Rocío Avenida Manuel Siurot Seville Spain Withdrawn
Great Ormond Street Hospital for Children NHS Trust London United Kingdom Recruiting

More Otsuka Pharmaceutical Development & Commercialization, Inc. trials in the USA

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04782258 on ClinicalTrials.gov ↗ ← All trials in the USA