About this study
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that can affect children. In PNH, red blood cells break apart too soon. Red blood cells carry oxygen through the body. When these cells break too early, the body does not get enough oxygen. This can cause fatigue, weakness, pain, anemia, and a higher risk of blood clots.
Some treatments can help, but they do not work well for all children. Some children still need blood transfusions. Other treatments require regular clinic visits, which can be hard for families. Doctors want to find a treatment that works well and is easier for children to take.
Iptacopan is a pill that has helped many adults with PNH. It can raise hemoglobin, which helps carry oxygen in the blood. Higher hemoglobin can help children feel less tired and need fewer transfusions. This study tests iptacopan in children and teens to see if it is safe and how well it works. All participants take iptacopan as a capsule or as a small tablet that can be swallowed whole.
The study can last up to 52 weeks. Doctors check blood tests and watch for side effects. They also check heart rate, blood pressure, and signs of infection. Because this medicine affects the immune system, children must get certain vaccines before joining the study to help prevent serious infections.
Doctors also look at anemia during the study. They measure hemoglobin levels and see if red blood cells are still breaking apart too early. They track how many blood transfusions each child needs. Some older children answer questions about tiredness and daily activities to show how they feel during treatment.
What we learn from this study may help guide future treatments for children with PNH and make care easier for families.
Eligibility overview
- Has paroxysmal nocturnal hemoglobinuria (PNH)
- Is 2–17 years old
- Has low hemoglobin levels or signs of anemia related to PNH
- Has received PNH treatment before or is new to treatment
- Is up to date on required vaccines before starting the study