Abstract
VALIANT: A Randomized, Multicenter, Double-Blind, Placebo (PBO)-Controlled, Phase 3 Trial of Pegcetacoplan for Patients with Native or Post-transplant Recurrent Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN): SA-OR92
Journal of the American Society of Nephrology, Vol.35(10S)
10/2024
DOI: 10.1681/ASN.2024qdwvz5bg
Abstract
Background:
VALIANT (NCT05067127) is a double-blind, PBO-controlled trial investigating the efficacy/safety of pegcetacoplan (PEG), a C3/C3b inhibitor, in adolescents (≥12 yrs) and adults with native or post-transplant recurrent C3G or primary IC-MPGN.
Methods:
Patients (pts) received PEG (SC infusion 2x/wk) or PBO (randomized 1:1) for 26 wks. The primary endpoint was log-transformed ratio of uPCR at wk 26 vs. baseline to measure proteinuria reduction vs. PBO.
Results:
124 pts were randomized to PEG (n=63) or PBO (n=61). The primary endpoint was met:68.3% (95% CI –76.3, –57.7) uPCR reduction in PEG vs. PBO arms at wk 26 (p<0.0001; Table). Results were consistent across all subgroups (disease type, age, and transplant status). Robust reductions in C3c staining and clinically meaningful eGFR stabilization were observed with PEG vs. PBO (Table). Treatment-emergent AE frequency and severity were similar between arms. None of the 4 serious infections (3 PEG; 1 PBO) were attributed to encapsulated bacteria. 1 death occurred in the PEG arm due to COVID-19 pneumonia (unrelated to PEG).
Conclusion:
PEG, a C3/C3b inhibitor, is the first therapy to achieve significant and clinically meaningful reductions in proteinuria (68.3% vs. PBO) and C3c staining and eGFR stabilization, compared with PBO in pts ≥12 yrs with C3G or primary IC-MPGN and was well tolerated.
Details
- Title: Subtitle
- VALIANT: A Randomized, Multicenter, Double-Blind, Placebo (PBO)-Controlled, Phase 3 Trial of Pegcetacoplan for Patients with Native or Post-transplant Recurrent Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN): SA-OR92
- Creators
- Carla M. Nester - University of IowaAndrew S. Bomback - Columbia University Irving Medical CenterMaría Gema Ariceta Iraola - Vall d'Hebron Hospital UniversitariYahsou Delmas - Centre Hospitalier Universitaire de BordeauxBradley P. Dixon - University of Colorado Anschutz Medical CampusDaniel P. Gale - University College LondonLarry A. Greenbaum - Emory UniversitySeung Hyeok Han - Yonsei UniversityNicole Isbel - University of QueenslandChristoph Licht - Hospital for Sick ChildrenAntonio Mastrangelo - Fondazione IRCCS Ca' Granda Ospedale Maggiore PoliclinicoMasashi Mizuno - Nagoya UniversityMaria Izabel Neves de Holanda - Hospital Geral de BonsucessoMatthew C. Pickering - Imperial College LondonGiuseppe Remuzzi - Mario Negri Institute for Pharmacological ResearchNicole Van De Kar - Amalia KinderziekenhuisMarina Vivarelli - Bambino Gesù Children's HospitalPatrick D. Walker - Arkana LaboratoriesDean Wallace - Royal Manchester Children's HospitalDaniel Zecher - Krankenhaus Barmherzige BrüderLi Li - Apellis PharmaceuticalsZhongshen Wang - Apellis PharmaceuticalsLuis López Lázaro - Swedish Orphan Biovitrum AB, Stockholm, Stockholm, SwedenJohan Szamosi - Swedish Orphan BiovitrumFadi Fakhouri - University Hospital of Lausanne
- Resource Type
- Abstract
- Publication Details
- Journal of the American Society of Nephrology, Vol.35(10S)
- Publisher
- AMER SOC NEPHROLOGY
- DOI
- 10.1681/ASN.2024qdwvz5bg
- ISSN
- 1046-6673
- eISSN
- 1533-3450
- Language
- English
- Date published
- 10/2024
- Academic Unit
- Nephrology, Dialysis and Transplantation; Internal Medicine; Stead Family Department of Pediatrics
- Record Identifier
- 9984740958002771
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