Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

This study is looking for adults aged 18 or older who have been diagnosed with atypical Haemolytic Uremic Syndrome (aHUS). It's important that your doctors have already ruled out other similar conditions.

You should also have been taking an anti-C5 antibody treatment for at least three months, and your doctor needs to confirm that this treatment has been working well for you. This means your blood tests (like platelet count and an enzyme called LDH) should be normal, and your kidney function should be stable.

However, you can't join if you've had a relapse of your aHUS while on your current medication, or if you have very poor kidney function. Also, if you have certain infections (like meningitis or pneumonia) or have had organ transplants, you wouldn't be able to take part. You'll also need to be up-to-date with specific vaccinations.

If you decide to participate, the study will involve several stages. First, there's a "screening period" that can last up to 14 weeks. During this time, doctors will do tests to make sure you meet all the requirements for the study. After screening, you'll enter the "Core Treatment period," where you'll switch from your current anti-C5 antibody treatment to iptacopan. This period lasts for 12 months. Following that, there's an optional "Extension Treatment period," which also lasts for 12 months.

Throughout the study, you'll have regular hospital visits for appointments, blood tests, and other checks to see how the new medication is affecting your aHUS and your overall health. These visits will help the doctors understand how well iptacopan is working and if there are any side effects. You'll be taking the iptacopan medication as directed by the study team.