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Two New Clinical Trials for Pediatric Kidney Diseases Open at UPMC Children’s

January 20, 2022

Two new clinical trials for pediatric patients with chronic kidney disease and polycystic kidney disease are now open and recruiting patients at UPMC Children’s Hospital of Pittsburgh in the Division of Pediatric Nephrology. Division clinical director Michael L. Moritz, MD, is the site principal investigator for both of the multicenter trials. In addition to serving as clinical director of the Division, Dr. Moritz is the medical director of pediatric dialysis and pediatric kidney transplantation at UPMC Children’s. Dr. Moritz also is an internationally respected expert in the fields of sodium disorders and water metabolism.

Treating Anemia in Patients with Chronic Kidney Disease

Anemia in patients with chronic kidney disease (CKD) is a common occurrence, particularly in more advanced stages of kidney dysfunction, and can lead to complications within the heart and a higher risk for stroke.1

A multicenter, open-label study of the oral medication vadadustat is being conducted to determine the safety and efficacy of a once daily dose for treating anemia in CKD after converting from an erythropoiesis stimulating agent. Vadadustat is an hypoxia-inducible factor (HIF) inhibitor that can stimulate red blood cell production.

This phase 3 study will follow 4 cohorts of pediatric patients based on their age: ≥ 12 to < 17 years; ≥ 6 to < 12 years; ≥ 2 to < 6 years; and ≥ 4 months to < 2 years.

New Drug Trial for Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc., a phase 3b multicenter open-label trial will test the safety, efficacy, and tolerability of the drug tolvaptan for the treatment of autosomal recessive polycystic kidney disease (ARPKD). Tolvaptan is a vasopressin antagonist that is often used to treat hyponatremia in the presence of heart failure, autosoma dominant polycystic kidney disease in adults, and several other conditions.

Infants and children between the ages of 28 days and 18 years with a clinical diagnosis of ARPKD may be eligible to participate in the study. The trial has two arms to test the use of both an oral suspension at varying doses and a tablet form of the drug in split dose regimens.

Prior studies of tolvaptan in adults with autosomal dominant polycystic kidney disease, a disease closely related to ARPKD have shown efficacy by slowing down the progressive decline of kidney function. This new trial will be the first to test tolvaptan in children with ARPKD. The study will examine the incidence of adverse events and other measures.

Additional information and trial protocol details are available at, Identifier: NCT04782258.

Patient Referrals and Additional Information

To refer a patient to UPMC Children’s for either of the clinical trials above, please contact the Division of Pediatric Nephrology at UPMC Children’s Hospital of Pittsburgh at 412-692-5182.


1. 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 Autosomal Recessive Polycystic Kidney Disease (ARPKD). Identifier: NCT047892258.

2. National Institute of Diabetes and Digestive and Kidney Disorders.

3. Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease. Identifier: NCT05082571.