We are studying the safety and tolerability of iptacopan in children with PNH. The trial will also evaluate its effects on hemoglobin levels and the need for blood transfusions.
Health conditions and diseases that the clinical trial is designed to study and treat.
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disorder characterized by the destruction of red blood cells, leading to symptoms such as dark urine, fatigue, and abdominal pain, especially during the night.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.
