Report
CT-286: Results of the PEGASUS Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria
Carlos de Castro, Peter Hillmen, Jeff Szer, Ilene Weitz, Alexander Röth, Britta Höchsmann, Jens Panse, Kensuke Usuki, Morag Griffin, Jean-Jacques Kiladjian, Hisakazu Nishimori, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, Cedric Francois, Federico Grossi, Antonio Risitano, Régis Peffault de Latour
Clinical Lymphoma, Myeloma and Leukemia | Elsevier BV | Published : 2020
Abstract
Context Anemia persists in up to 72% of patients with paroxysmal nocturnal hemoglobinuria (PNH) despite the inhibition of C5 with eculizumab. Pegcetacoplan, a C3 inhibitor, has the potential to control both intravascular (IVH) and extravascular hemolysis (EVH). Objective The phase 3, randomized, open-label, controlled PEGASUS trial (NCT03500549) assessed efficacy and safety of pegcetacoplan compared to eculizumab in patients with PNH. Patients, Interventions, Main Outcome Measures Patients aged ≥18 years with PNH and hemoglobin <10.5 g/dL completed a 4-week run-in period with pegcetacoplan plus eculizumab before 1:1 randomization to monotherapy with pegcetacoplan (n=41) or eculizumab (n=39)...
View full abstract