A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Trial Parameters
Brief Summary
This is a multicenter, open-label, Phase Ib clinical trial designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of the humanized monoclonal antibody EA5 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Eligibility Criteria
Inclusion Criteria: * Male or female subjects aged ≥18 years. * Body weight between 40 kg and 100 kg (inclusive) at screening. * Patients diagnosed with PNH, confirmed by flow cytometry demonstrating a PNH clone size (glycosylphosphatidylinositol-anchored protein-deficient granulocytes or monocytes) of ≥10% in peripheral blood, and meeting one of the following criteria: * a) Previously naive to complement inhibitor therapy; or * b) Previously treated with a complement inhibitor, which has been discontinued for ≥5 half-lives prior to screening. * Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal (ULN) at screening. * Presence of one or more of the following PNH-related signs or symptoms within 3 months prior to screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin \<10 g/dL), history of major thrombotic event (including thrombosis), dysphagia, or erectile dysfunction; or a history of packed red blood cell (pRBC) transf