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)
Start Date
1/23/2023
Completion Date
8/14/2028
Summary
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
Detailed Description
This study is a multinational, multicenter, open-label, non-randomized trial. The study consist of three periods: Screening Period, Treatment period and Follow-up period. 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 24 months over the course of the study. The overall trial duration is expected to be approximately 4.1 years.
Eligibility Criteria
Age Range: No minimum to 18 years
Interventions
Tolvaptan Suspension
Tolvaptan Tablets
Conditions
Locations
Children's National Medical Center
Washington D.C., District of Columbia 20010
United States
Emory University Hospital
Atlanta, Georgia 30322
United States
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
Chicago, Illinois 60611
United States
Riley Hospital for Children
Indianapolis, Indiana 46202-5119
United States
Children's Hospital - New Orleans
New Orleans, Louisiana 70118
United States
Johns Hopkins Pediatric Specialty Clinic
Baltimore, Maryland 21287
United States
C.S. Mott Children's Hospital
Ann Arbor, Michigan 48109-5000
United States
Mayo Clinic - Rochester
Rochester, Minnesota 55905
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229-3039
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania 15213
United States
Primary Children's Hospital
Salt Lake City, Utah 84113
United States
Université Catholique De Louvain And Cliniques St Luc
Brussels, Brussels Capital 1200
Belgium
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen 9000
Belgium
UZ Leuven
Leuven, Vlaams Brabant 3000
Belgium
Universitätsklinikum Köln
Cologne, North Rhine-Westphalia 50937
Germany
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, Masovian Voivodeship 04-730
Poland
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
Bialystok, 15-274
Poland
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
Esplugues de Llobregat, Barcelona 8950
Spain
Hospital Universitari Parc Tauli
Sabadell, Barcelona 08208
Spain
Hospital Universitari Vall D Hebron
Barcelona, 8035
Spain
Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
Seville, 41013
Spain
Great Ormond Street Hospital for Children NHS Trust
London, WC1N 3JH
United Kingdom