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 ARPKD

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 18 months over the course of the study.

The overall trial is expected to last approximately 3.5 years.

Eligibility Criteria

  • Ages 28 days to 18 years old
  • 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
    • a history of oligohydramnios or anhydramnios in utero.
  • 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.

Additional inclusion/exclusion criteria may apply

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



Participation will continue over 18 months and include oral administration of tolvaptan either once or twice daily depending on age. Outcome measurements will be taken at enrollment, 1 month, 6 months, 12 months, 18 months, and up to 7 days past last dose of tolvaptan.


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

Children’s Hospital of Pittsburgh of UPMC
Pittsburgh, PA 15213

Riley Hospital for Children
Indianapolis, IN 46202

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

Chicago, IL 60611

Primary Children’s Hospital
Salt Lake City, UT 84113