Abstract
Long-Term Safety and Efficacy of Pegcetacoplan in Patients with C3 Glomerulopathy or Primary Immune-Complex Membranoproliferative Glomerulonephritis: The Long-Term VALE Extension Study: SA-PO801
Journal of the American Society of Nephrology, Vol.35(10S)
10/2024
DOI: 10.1681/ASN.20243n9dsy1s
Abstract
Background:
C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) are rare diseases caused by complement overactivation leading to excessive deposition of C3 breakdown products in the kidney, causing inflammation, renal damage, and renal failure. Pegcetacoplan (PEG; C3/C3b inhibitor) blocks overactivation of alternative and classical complement pathway by inhibiting both C3 and C5 convertases (Figure). In the phase 2 trials, PEG demonstrated clinical benefit and was well tolerated. The Phase 3 VALIANT (NCT05067127) trial, in patients with C3G or primary IC-MPGN in native kidney or post-transplant, will further evaluate the safety and efficacy of PEG. Here we describe VALE (NCT05809531), a phase 3 extension study designed to evaluate long-term efficacy and safety of PEG in C3G and primary IC-MPGN.
Methods:
Patients ≥12 years old who completed VALIANT through week 52 and achieved clinical benefit will continue to receive twice-weekly doses of PEG for a minimum of approximately 2.5 years. The primary efficacy endpoint is the log-transformed urine protein-to-creatinine ratio vs baseline. Secondary endpoints will evaluate changes in estimated glomerular filtration rate, proteinuria, and patient-reported outcomes, and disease progression defined by a composite clinical outcome. Safety endpoints will assess treatment-emergent adverse events.
Results:
Not available.
Conclusion:
The VALE extension will assess long-term efficacy and safety of PEG in C3G and primary IC-MPGN.
Details
- Title: Subtitle
- Long-Term Safety and Efficacy of Pegcetacoplan in Patients with C3 Glomerulopathy or Primary Immune-Complex Membranoproliferative Glomerulonephritis: The Long-Term VALE Extension Study: SA-PO801
- Creators
- Carla M. Nester - University of IowaMaría Gema Ariceta Iraola - Universitat Autònoma de BarcelonaAnwesha Mukherjee - Apellis PharmaceuticalsLi Li - Apellis PharmaceuticalsJohan Szamosi - Swedish Orphan BiovitrumLuis López Lázaro - Swedish Orphan Biovitrum AB, Stockholm, Stockholm, Sweden
- Resource Type
- Abstract
- Publication Details
- Journal of the American Society of Nephrology, Vol.35(10S)
- Publisher
- AMER SOC NEPHROLOGY
- DOI
- 10.1681/ASN.20243n9dsy1s
- ISSN
- 1046-6673
- eISSN
- 1533-3450
- Language
- English
- Date published
- 10/2024
- Academic Unit
- Internal Medicine; Stead Family Department of Pediatrics; Nephrology, Dialysis and Transplantation
- Record Identifier
- 9984740958702771
Metrics
3 Record Views