Genetic Testing   

Pediatric Researchers Serve as New Jersey’s Leaders for the Study of Rare Diseases Diagnosed Through Genetic Testing

Four clinical trials will provide Hackensack Meridian Children’s Health patients access to promising new treatments

Genetic Testing

Pediatric nephrology researchers at Hackensack Meridian Children’s Health — which is ranked No. 34 in the nation for pediatric nephrology by U.S. News & World Report — are participating in four medication clinical trials for children and young adults with kidney disease.

As a network, Joseph M. Sanzari Children’s Hospital and K. Hovnanian Children’s Hospital, are leaders in research breakthroughs, and the treatment and diagnosis of rare pediatric kidney conditions. Beginning with the work done under the leadership of Dr. Kenneth Lieberman, chief of the Pediatric Nephrology at Joseph M. Sanzari, the network has become a leader in the diagnostics of rare kidney diseases using genetics.

The international, multicenter trials are offered at Joseph M. Sanzari Children’s Hospital and provide young patients with access to promising treatments years before they become widely available. Clinical trials include:

  • VALIANT trial. Joseph M. Sanzari Children’s Hospital is currently the only site in New Jersey offering this study, which aims to determine the effectiveness of subcutaneously infused medication pegcetacoplan when compared to placebo in reducing high levels of protein in the urine. The trial is open to patients aged 12 years and older with C3 glomerulopathy and immune-complex membranoproliferative glomerulonephritis.
  • COMMUTE-p trial. This study aims to study the effectiveness of the intravenous medication crovalimab in pediatric patients with atypical hemolytic uremic syndrome. Joseph M. Sanzari Children’s Hospital is currently the only site in New Jersey to offer this clinical trial.
  • INShore trial. The goal of this trial is to study the efficacy, safety, and pharmacokinetics/pharmacodynamics of intravenous medication obinutuzumab compared with mycophenolate mofetil (MMF) in children and young adults aged 2-25 with frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome.
  • VX-147 trial. This study will evaluate the safety, efficacy, tolerability, and pharmacokinetics of oral medication VX-147 in patients aged 18 and older with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

Learn more about our advancements in pediatric nephrology.

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