A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Who is this study for? Patients with Autosomal Recessive Polycystic Kidney Disease (ARPKD)
What treatments are being studied? Tolvaptan (OPC-41061)
Status: Recruiting
Location: See all (18) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
Eligibility
Participation Requirements
Sex: All
Minimum Age: 28 days
Maximum Age: 2 months
Healthy Volunteers: f
View:
• Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
• Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
‣ Nephromegaly (\> 2 standard deviations from age-appropriate standard via ultrasound)
⁃ Multiple renal cysts
⁃ History of oligohydramnios or anhydramnios
• Ability for parent or 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.
Locations
United States
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Georgia
Emory University Hospital
NOT_YET_RECRUITING
Atlanta
Illinois
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
RECRUITING
Chicago
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
Utah
Primary Children's Hospital
WITHDRAWN
Salt Lake City
Other Locations
Belgium
Universitair Ziekenhuis Gent
RECRUITING
Ghent
UZ Leuven
RECRUITING
Leuven
Germany
Universitätsklinikum Köln
WITHDRAWN
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WITHDRAWN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WITHDRAWN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 Universitario Virgen del Rocio
WITHDRAWN
Seville
United Kingdom
Great Ormond Street Hospital for Children NHS Trust