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Pegcetacoplan for 52 Weeks Maintains Proteinuria Reduction and eGFR Stabilization in Pediatric Patients: Phase 3 VALIANT Subgroup Analysis: FR-PO0693
Abstract   Peer reviewed

Pegcetacoplan for 52 Weeks Maintains Proteinuria Reduction and eGFR Stabilization in Pediatric Patients: Phase 3 VALIANT Subgroup Analysis: FR-PO0693

Larry A. Greenbaum, Carla M. Nester, María Gema Ariceta Iraola, Bradley P. Dixon, Yael Borovitz, Christoph Licht, Antonio Mastrangelo, Nabil Z. Melhem, Naoya Fujita, Nicole Van De Kar, …
Journal of the American Society of Nephrology, Vol.36(10S)
10/2025
DOI: 10.1681/ASN.2025w64wx1r9

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Abstract

Background: C3 glomerulopathy (C3G) and primary immune-complex mediated membranoproliferative glomerulonephritis (IC-MPGN) are often diagnosed in childhood. In the 26-week randomized controlled period (RCP) of VALIANT (phase 3; NCT05067127), pegcetacoplan (C3/C3b inhibitor) for C3G and primary IC-MPGN led to significant proteinuria reduction vs placebo and estimated glomerular filtration rate (eGFR) stabilization for adolescents (12–17 years) and the overall population. In the following 26-week open-label period (OLP), all patients received pegcetacoplan. Methods: 28 and 27 adolescents were randomized to pegcetacoplan or placebo, respectively; 27 and 25 adolescents, respectively, entered the OLP. Efficacy end points included changes from baseline in proteinuria (measured as urine protein-to-creatinine ratio [UPCR]) and eGFR. Treatment-emergent adverse events were noted. Results: Adolescents who received pegcetacoplan for 52 weeks had sustained proteinuria decrease (mean [95% CI] UPCR change from baseline: week 26, –73.6% [–83.1, –58.6]; week 52, –71.8% [–82.9, –53.6]) (Figure). Results for placebo-to-pegcetacoplan patients in the OLP were consistent with proteinuria decreases in the RCP for the pegcetacoplan group. Adolescents achieved stable eGFR throughout VALIANT (least squares mean [SE] change from baseline: week 26, +0.7 [3.6] mL/min/1.73 m2; week 52, +3.2 [4.1] mL/min/1.73 m2). No new safety signals were identified. Conclusion: Clinical efficacy of 52 weeks of pegcetacoplan treatment for adolescents was consistent with the overall population of VALIANT, confirming its sustained benefit in broad patient populations.

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