Post-Marketing Safety and Effectiveness Study of Iptacopan in Korean Adults with Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy

What this Trial Is About

Researchers are conducting a post-marketing surveillance study in South Korea to evaluate the safety and effectiveness of iptacopan for treating patients diagnosed with either Paroxysmal Nocturnal Hemoglobinuria PNH or C3 Glomerulopathy C3G. The study collects real-world data from medical records of Korean patients receiving iptacopan under routine clinical care as part of the Risk Management Plan. This observational study aims to understand how iptacopan performs in everyday treatment settings for these conditions. Participants include adults diagnosed with PNH or C3G who have received or will receive iptacopan according to the approved Korean labeling. The study monitors two patient groups those treated for PNH and those treated for C3G. Patients must have received recommended vaccinations before starting iptacopan. Treatment is not assigned by the study but follows routine medical practice, and all dosing and administration decisions are made by the treating physicians. During the study, researchers collect data on adverse events and drug reactions over up to two years. Laboratory measurements such as hemoglobin, lactate dehydrogenase, blood cell counts, kidney function tests, and urine protein levels are collected at the start and at 24 weeks to assess changes. Investigators also evaluate overall patient improvement at 24 weeks. Participants provide informed consent, and their health information from medical records is used to assess treatment outcomes and safety in routine clinical care.

A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy