A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With ARPKD

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This study involves an investigational drug approved by the FDA for the treatment of autosomal dominant PKD. The purpose of the 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).  The trial will be the first trial of tolvaptan in a pediatric ARPKD population.

Participants in this study will be administered tolvaptan orally or via nasogastric tube either once or twice a day (depending on age) for 2 years and closely monitored over the course of the study.


Your child may be eligible for this study if they:
● Are between 28 days and <12 weeks of age
● Have been diagnosed with ARPKD


Exclusion Criteria:
● Premature at birth (<32 weeks gestational age)
● Have had a kidney transplant
● Abnormal liver function tests
● Require ventilator support


Once complete, participants may be eligible for a second trial to continue to evaluate tolvaptan in ARPKD until the age of 18.


If you’re interested in participating, contact: Lindsay Getz @ 919.797.9591 or lindsay.getz@paidion.com