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)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) — From baseline to post-treatment after 24 months or EoTx
Number of participants with TEAEs will be assessed from Baseline to post-treatment after 24 months or End of Treatment (EOTx). The EOTx visit applies to participants who discontinue IMP before Month 24. TEAEs are defined as Adverse Events (AEs) with an onset date on or after the start of Investigational Medicinal Product (IMP) treatment. TEAEs are also events continuous from baseline which worsened, became serious, were IMP related, or resulted in death, discontinuation, interruption, or reduction of IMP. An AE is defined as any untoward medical occurrence in a clinical trial subject administered an IMP and which does not necessarily have a causal relationship with this treatment.
Trial sites (23)
| Facility | City | Region | Status |
|---|---|---|---|
| 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| Emory University Hospital | Atlanta | Georgia | Withdrawn |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Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology | Chicago | Illinois | Withdrawn |
| Riley Hospital for Children | Indianapolis | Indiana | Withdrawn |
| Children's Hospital - New Orleans | New Orleans | Louisiana | Withdrawn |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| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | Withdrawn |
| Primary Children's Hospital | Salt Lake City | Utah | Withdrawn |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 UK
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 UK