Kidney
Clinical Trial of New Medication to Treat Children with Rare Kidney Diseases
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Feb 15, 2022 - 7:00:02 AM

(HealthNewsDigest.com) - EDISON, N.J. – Hackensack Meridian Children’s Health is the only pediatric health care network in New Jersey undertaking a new study that examines the effectiveness of a drug called sparsentan to treat rare kidney diseases in children.  Chief pediatric nephrologists and Hackensack Meridian School of Medicine professors, Ken Lieberman, M.D. and Guillermo Hidalgo, M.D., F.A.S.N, are the study’s principal investigators.

The study, called “Study of Sparsentan Treatment in Pediatrics with Proteinuric Glomerular Diseases (EPPIK),” is open to pediatric nephrology patients throughout New Jersey at both Hackensack Meridian Children’s Hospitals - Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center and K. Hovnanian Children’s Hospital at Jersey Shore University Medical Center.

This EPPIK study  is investigating the use of the medication to treat three specific proteinuric diseases — steroid-resistant nephrotic syndrome*, Alport syndrome**, and IgA nephropathy***. These diseases typically develop in children or young adults, cause progressive loss of kidney function, and result in end-stage kidney disease.

Proteinuric kidney diseases are rare and result in a condition called proteinuria, or too much protein in the urine. High levels of protein in the urine can affect kidney function. And because proteins are needed to build muscle and bone, regulate the amount of fluid in blood, combat infection and repair tissue in the body, it isn’t healthy for proteins to enter the urine and leave the body. Up until now the three conditions being studied have been treated with other classes of medications, including immunosuppressive drugs. At times for extended periods of time.

Hackensack Meridian Children’s Health researchers have participated in two previously published studies of sparsentan, which is manufactured by Travere Therapeutics. This latest EPPIK trial is the first to be a 100-percent pediatric study. This trial joins our other trials currently enrolling or in the process of being opened for: anemia in chronic kidney disease and atypicalaytpical hemolytic-uremic syndrome.

“These conditions have the likelihood of progressing to irreversible kidney failure, and previous treatments have not been successful,” said Ken Lieberman, M.D., chief of Pediatric Nephrology, professor, Hackensack Meridian School of Medicine, and EPPIK principal investigator at Joseph M. Sanzari Children’s Hospital. “The EPPIK trial gives pediatric kidney disease patients throughout New Jersey access to a promising new treatment before it is available to the public.”

“Living with a rare, complex kidney disease is challenging not just for children, but for their families, too,” said Guillermo Hidalgo, M.D., F.A.S.N, professor, Hackensack Meridian School of Medicine, section head of pediatric nephrology and principal investigator at K. Hovnanian Children’s Hospital.

“Through clinical trials like EPPIK, our goal is to advance the standard of care for proteinuric kidney disease and offer hope through novel treatments.”

“As the only children’s healthcare network in New Jersey that is participating in the EPPIK trial, Hackensack Meridian Children’s Health is establishing itself as a premier center for the treatment of rare pediatric kidney disease,” said Judy Aschner, M.D., physician-in-chief, Pediatrics, Hackensack Meridian Children’s Health and Joseph M. Sanzari Children’s Hospital, and professor, Pediatrics, Hackensack Meridian School of Medicine.

“Our researchers are committed to improving the quality of life and prognosis for children and adolescents who are living with these conditions,” said Harpreet Pall,M.D., MBA, CPE, chair, Pediatrics, K. Hovnanian Children’s Hospital, and chair and professor, Pediatrics at Hackensack Meridian School of Medicine.

*About Steroid-Resistant Nephrotic Syndrome (SRNS)

• Can be caused by genetic abnormalities or immune system dysfunction
• Rare and seen predominantly in males.
• Holds a significant risk for speedy progression from chronic kidney disease to end-stage renal disease.
• Leading cause of end-stage kidney disease
• Nephrotic syndrome (NS) is a clinical syndrome characterized by massive proteinuria, hypoalbuminemia, peripheral edema, and hyperlipidemia

**About Alport Syndrome

• Stems from the mutations in COL4A3, COL4A4, COL4A5, which play a major role in structures called glomeruli.
• Glomeruli are clusters of special blood vessels that remove waste products and water from the blood and create urine.
• Syndrome attacks the glomeruli by damaging the tiny blood vessels in kidneys.
• Leads to loss of this organ’s functions which in turn leads to eye problems and hearing loss.

***About IgA nephropathy also known as Berger’s Disease (Immunoglobin A)

• Caused by buildup of an antibody called immunoglobin A
• Inflammation damages the kidney which later on affects the processes that filter waste from the body’s blood.
• Disease is more common in men than it is in women

About the Pediatric Nephrology Program

As a network, Hackensack Meridian Children’s Health is a  leader in research breakthroughs and exceptional outcomes for children with kidney disease and hypertension.The pediatric nephrology program at Hackensack Meridian Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center and K. Hovnanian Children’s Hospital at Jersey Shore University Medical Center, plays a critical role in understanding and improving outcomes for children with renal disease. Both children’s hospitals serve as clinical coordinating centers for the world’s largest registry for crescentic glomerulonephritis, and an expanded acute kidney injury program at K. Hovnanian includes new interdisciplinary care that provides vital dialysis services. The comprehensive program also offers genetic testing and counseling. In 2021, Sanzari, which is home to the network’s pediatric transplant program, performed a record number of kidney transplants. Namrata G. Jain, M.D., previously the medical director of the Pediatric Transplant at New York-Presbyterian Hospital/Columbia, in 2021 became the new medical director of pediatric kidney transplant.

Hackensack Meridian Children’s Health

Hackensack Meridian Children’s Health provides the most comprehensive and highest level of quality care to young patients in the state of New Jersey. The children’s network is comprised of two children’s hospitals – Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center in Hackensack and K. Hovnanian Children’s Hospital at Jersey Shore University Medical Center in Neptune – and a large network of pediatric subspecialists and pediatricians. Hackensack Meridian Children’s Health hospitals are the only two in New Jersey to be ranked among the top 50 in the nation for pediatric cancer care by U.S. News and World Report. Joseph M. Sanzari Children’s Hospital also ranks among the top 50 in the nation for pediatric neurology and neurosurgery. Visit www.hackensackmerdianhealth.org/kids for more information about Hackensack Meridian Children’s Health.