Trial Studies Safety, Efficacy of Medication for Ultra-Rare C3 Glomerulopathy

Hackensack Meridian Children’s Health is participating in the Phase 3 international VALIANT clinical trial for C3 glomerulopathy. The VALIANT trial is evaluating the efficacy and safety of the FDA-approved medication pegcetacoplan compared with placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN). 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.

Prior to the FDA approval of pegcetacoplan, there was no available, effective treatment for C3G — an ultra-rare condition that affects 1-2 people out of a million in the U.S. each year. Pegcetacoplan is administered via subcutaneous infusion twice weekly for 6 months.

The goal of the study is to determine whether pegcetacoplan improves the urine protein-to-creatinine ratio compared with placebo.

“For the past 20 years, our pediatric nephrology team has been participating in revolutionary clinical trials that address unmet needs for treating rare or ultra-rare disorders of the kidney,” said Dr. Lieberman. “Through our clinical trials, we bring new treatments to the point of FDA approval, provide earlier access to promising treatments for patients at HMCH, and advance treatment options for all pediatric nephrologists and the children under their care.”

Learn more about our advancements in pediatric nephrology.