Trial Studies Safety, Efficacy of First Medication for Relapsing Pediatric Nephrotic Syndrome

Hackensack Meridian Children’s Health enrolled the first American patient and is the top enroller worldwide in a Phase 3 clinical trial studying the safety and efficacy of a revolutionary medication to treat relapsing nephrotic syndrome. Kenneth V. Lieberman, M.D., chief of Pediatric Nephrology, and pediatric nephrologist Guillermo Hidalgo, M.D., are serving as co-investigators on the clinical trial. The international INShore clinical trial is studying the efficacy and safety of FDA-approved medication obinutuzumab compared with mycophenolate mofetil (MMF) in children and young adults who have been diagnosed with frequently relapsing nephrotic syndrome or steroid-dependent nephrotic syndrome. Although other medications have been used to treat nephrotic syndrome for years, obinutuzumab is the first medication approved specifically to treat relapsing nephrotic syndrome that was diagnosed in childhood. It is administered by intravenous infusion in four doses over a six-month period. The goal of the study is to determine the percentage of patients achieving sustained complete remission at one year.

“Relapsing nephrotic syndrome is a relatively common condition in a pediatric nephrology practice, but before obinutuzumab, there were no medications specifically indicated for the condition,” said Dr. Lieberman, who co-authored an abstract about this research that has been submitted to the World Congress of Nephrology. “Now, pediatric nephrologists have a new option to help manage frequent nephrotic syndrome relapses, with the goal of reducing relapse frequency and improving quality of life for patients.”

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