Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial
Antonio M. Risitano, Alexander Röth, Juliette Soret, Camilla Frieri, Flore Sicre de Fontbrune, Luana Marano, Ferras Alashkar, Lina Benajiba, Serena Marotta, Izabela Rozenberg, Julie Milojevic, Peter End, Prasanna Kumar Nidamarthy, G. Junge, Régis Peffault de Latour
Topics & Concepts
MedicineEculizumabParoxysmal nocturnal hemoglobinuriaOpen labelHaemolysisInternal medicineClinical trialImmunologyAntibodyComplement systemComplement system in diseasesBlood groups and transfusionPlatelet Disorders and Treatments