Abstract: INFO17-SA
Real-World Effectiveness and Safety of Pegcetacoplan in Patients with C3 Glomerulopathy or Primary Immune Complex-Membranoembranoproliferative Glomerulonephritis: PRISM-C3, a Multicountry Phase 4 Study
Session Information
- Informational Posters - 3
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- No subcategory defined
Authors
- Kavanagh, David, Newcastle University, Newcastle upon Tyne, United Kingdom
- Pickering, Matthew C., Imperial College London Department of Immunology and Inflammation, London, England, United Kingdom
- Cook, Terence, Imperial College London Department of Immunology and Inflammation, London, England, United Kingdom
- Vivarelli, Marina, Laboratory of Nephrology and Clinical Trial Center, IRCCS Bambino Gesu Children's Hospital, Rome, Italy
- Le Quintrec, Moglie, Centre Hospitalier Universitaire de Lapeyronie, Montpellier, France
- Fremeaux Bacchi, Veronique, Department of Immunology Biology, Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Paris, France
- Taliadouros, Ginny, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Surova, Elena, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Webb, Nicholas J., Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Fakhouri, Fadi, Centre Hospitalier Universitaire Vaudois, Lausanne, VD, Switzerland
Description
C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (pIC-MPGN) are rare, chronic kidney diseases, driven by C3 dysregulation resulting in progressive kidney damage and high risk of kidney failure. Pegcetacoplan, a C3/C3b inhibitor, has demonstrated clinical efficacy (proteinuria reduction, estimated glomerular filtration rate stabilization, and clearance of glomerular C3 deposits) and a favorable safety profile in patients with C3G or pIC-MPGN in clinical trials; it is approved in the US and the EU for native disease and transplanted patients ≥12 years with C3G or pIC-MPGN. To complement clinical trial data, real-world evidence for pegcetacoplan is needed to support clinical decision making.
PRISM-C3 is an international (excluding US), multicenter, Phase 4 study to evaluate the real-world effectiveness of pegcetacoplan over at least 2 years. Starting in 2026, patients who have initiated or plan to initiate pegcetacoplan treatment for C3G or pIC-MPGN (native or post-transplant recurrent disease) outside of interventional clinical studies are being recruited. Patient data will be collected from medical records both retrospectively and prospectively (through routine clinic visits, observational aspect); blood and urine biomarker data will be collected prospectively at scheduled time points relating to the initiation or discontinuation of pegcetacoplan treatment (low interventional aspect). Biomarkers to be assessed include disease etiology markers, complement pathway biomarkers, and inflammatory and kidney injury biomarkers. The primary endpoint is the proportion of patients achieving a urine protein-to-creatinine ratio of <1 g/g after 6 months of pegcetacoplan treatment. Secondary objectives include evaluation of renal function and disease characteristics, biomarker changes, patient-reported outcomes, healthcare resource utilization, and safety.
The findings of this real-world study will support the long-term evaluation of the benefit-risk profile of pegcetacoplan in a broad population of patients with C3G or pIC-MPGN and may help to optimize evidence-based clinical practice. Assessing disease-specific biomarkers is a novel and valuable addition to evaluating the real-world effectiveness of pegcetacoplan.
Funding
- Swedish Orphan Biovitrum AB (Sobi)