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

Overview
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

 

Locations actively recruiting:

Children’s National Medical Center
Washington, D.C., 20010

Cincinnati Children’s Hospital
Cincinnati, OH, 44195

Locations pending study participation:

Emory University
Atlanta GA 30322

Mayo Clinic
Rochester, MN 55905

Cleveland Clinic
Cleveland, OH 44195