A recent publication in the Clinical Journal of the American Society of Nephrology presented outcomes of pegcetacoplan treatment in adolescents with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
C3G and IC-MPGN are rare kidney diseases characterized by damage to the glomeruli in the kidney due to abnormal activation of the complement system. Common signs and symptoms include hematuria, proteinuria, reduced glomerular filtration rate and increased creatinine levels, fatigue, and edema of the hands, feet, and ankles.
The VALIANT (NCT05067127) clinical trial is a randomized, placebo-controlled, double-blinded, multicenter study evaluating the safety and efficacy of pegcetacoplan in patients with C3G and IC-MPGN. Pegcetacoplan is a C3/C3b inhibitor that led to significant proteinuria reduction (greater than 68% versus placebo) and estimated glomerular filtration rate (eGFR) stabilization in native kidney and posttransplant recurrent C3G and IC-MPGN. This recent analysis provides results for adolescents, ages 12 to 17 years in VALIANT.
A total of 28 adolescents received pegcetacoplan and 27 received placebo. Consistent with the overall patient population in the VALIANT study, the majority of pegcetacoplan-treated adolescents achieved clinically meaningful urine protein-to-creatinine ratio (UPCR) reduction. 57% of adolescents in pegcetacoplan versus 4% in placebo arms achieved the composite endpoint of 15% or less eGFR reduction and 50% or greater UPCR reduction; and 50% or greater UPCR reduction was observed in 71% of pegcetacoplain-treated patients versus 4% in the placebo. eGFR was stable for pegcetacoplan-treated adolescents.
Three adolescents in each group experienced serious treatment-emergent adverse events with pyrexia in 1 pegcetacoplan-treated patient being considered treatment related. There were no infections caused by encapsulated bacteria.
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To learn more about C3G, IC-MPGN, and other rare kidney conditions, visit https://checkrare.com/diseases/kidney-and-urinary-diseases/