This clinical trial is focused on studying the long-term safety and tolerability of a medication called iptacopan in patients with a rare blood disorder known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The medication being tested, iptacopan, is taken in the form of hard gelatin capsules and is designed to help manage this condition.
The purpose of the study is to evaluate how safe and well-tolerated iptacopan is when used over a long period. Participants in this study are individuals who have already completed previous phases of clinical trials with iptacopan. The study will monitor participants for any side effects or health changes while they continue taking the medication. This includes regular check-ups and assessments to ensure the medication is working as intended without causing harm.
Throughout the study, participants will be observed for any adverse events, which are unexpected health issues that may arise. The study will also track the stability of hemoglobin levels, which are important for carrying oxygen in the blood, and check for any need for blood transfusions. Additionally, the study will look for any occurrences of breakthrough hemolysis, which is when red blood cells break down too quickly, and any major vascular events, which are serious issues related to blood vessels. The study aims to provide valuable information on the long-term use of iptacopan for managing PNH.



Czechia
France
Germany
Italy
Lithuania
Spain
The Netherlands