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

United States

Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).

The trial will be the first trial of tolvaptan in a pediatric ARPKD population.

Participants in this study will be assigned to tolvaptan for 24 months and closely monitored over the course of the study.

Eligibility Criteria

  • Ages: 28 days to 12 weeks old (Child)
  • Gender: All
  • 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 kidney cysts, and
    • 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.

For more information, please contact ARPKD.Pediatrics@otsuka-us.com



Participation will continue for 2 years and include the administration of tolvaptan orally or via NG tube once or twice daily depending on age. A parent questionnaire will be given immediately after and 15-20 minutes after first oral dose.


Locations actively recruiting:

Children’s National Medical Center
Washington, D.C., 20010
Contact: Krista Wink, Research Assistant
240-531-6619 kwink@childrensnational.org

Cincinnati Children’s Hospital
Cincinnati, OH, 44195

Locations pending study participation:

Cleveland Clinic
Cleveland, OH 44195

C.S. Mott Children’s Hospital
Ann Arbor, MI 48109

Emory University
Atlanta, GA 30322

Johns Hopkins Pediatric Specialty Clinic
Baltimore, MD 21287-0001

Mayo Clinic
Rochester, MN 55905

Northwestern University
Chicago, IL 60611

Primary Children’s Hospital
Salt Lake City, UT 84113-1103