A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Who is this study for? Patients with Autosomal Recessive Polycystic Kidney (ARPKD)
What treatments are being studied? Tolvaptan
Status: Recruiting
Location: See all (23) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
To evaluate the safety of tolvaptan in pediatric subjects with ARPKD
Eligibility
Participation Requirements
Sex: All
Minimum Age: 28 days
Maximum Age: 18
Healthy Volunteers: f
View:
• 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.
• 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.
Locations
United States
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Georgia
Emory University Hospital
WITHDRAWN
Atlanta
Illinois
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
RECRUITING
Chicago
Indiana
Riley Hospital for Children
WITHDRAWN
Indianapolis
Louisiana
Children's Hospital - New Orleans
WITHDRAWN
New Orleans
Maryland
Johns Hopkins Pediatric Specialty Clinic
RECRUITING
Baltimore
Michigan
C.S. Mott Children's Hospital
RECRUITING
Ann Arbor
Minnesota
Mayo Clinic - Rochester
RECRUITING
Rochester
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Cleveland Clinic
RECRUITING
Cleveland
Pennsylvania
Children's Hospital of Pittsburgh of UPMC
WITHDRAWN
Pittsburgh
Utah
Primary Children's Hospital
WITHDRAWN
Salt Lake City
Other Locations
Belgium
Université Catholique De Louvain And Cliniques St Luc
RECRUITING
Brussels
Universitair Ziekenhuis Gent
RECRUITING
Ghent
UZ Leuven
RECRUITING
Leuven
Germany
Universitätsklinikum Köln
RECRUITING
Cologne
Poland
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
WITHDRAWN
Bialystok
Instytut Pomnik - Centrum Zdrowia Dziecka
WITHDRAWN
Warsaw
Spain
Hospital Universitari Vall D Hebron
RECRUITING
Barcelona
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
RECRUITING
Esplugues De Llobregat
Hospital Universitari Parc Tauli
WITHDRAWN
Sabadell
Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
WITHDRAWN
Seville
United Kingdom
Great Ormond Street Hospital for Children NHS Trust
RECRUITING
London
Contact Information
Primary
Otsuka Call Center
Otsuka-ProfessionalServices@otsuka-us.com
844-687-8522
Time Frame
Start Date: 2023-01-23
Estimated Completion Date: 2028-08-14
Participants
Target number of participants: 20
Treatments
Experimental: 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.
Experimental: 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.
Related Therapeutic Areas
Sponsors
Leads: Otsuka Pharmaceutical Development & Commercialization, Inc.