Study of Iptacopan Treatment Effects on Adults With Paroxysmal Nocturnal Hemoglobinuria in China

What this Trial Is About

Researchers are conducting a real-world study to evaluate the impact of iptacopan on adult patients with paroxysmal nocturnal hemoglobinuria PNH in China. The study aims to assess treatment-related outcomes, disease management, and healthcare resource use, considering new standards for PNH care and the introduction of iptacopan. It includes patients who are either new to complement inhibitor therapy or those stable on C5 complement inhibitors like eculizumab. The study involves two patient groups one with PNH patients never treated with complement inhibitors who are starting iptacopan, and another with patients stable on C5 complement inhibitors who will switch to iptacopan after its approval in China. Treatments are oral capsules of LNP023 iptacopan, and patients must start iptacopan within 60 days of consenting. Participants must have documented vaccinations against Neisseria meningitidis and Streptococcus pneumoniae before starting treatment. Participants will be followed for at least 12 months, with assessments including hemoglobin levels, lactate dehydrogenase LDH, absolute reticulocyte count ARC, bilirubin, PNH clone size, and signs or symptoms related to PNH. Researchers will monitor treatment effects, adverse events, medication use, transfusions, and healthcare visits. Patient fatigue and work productivity will be evaluated using specific questionnaires, with data collected at baseline and regular intervals to understand iptacopans real-world impact on disease and quality of life.

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China