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 for adults with a rare kidney disease called atypical Hemolytic Uremic Syndrome (aHUS). If you're currently being treated with anti-C5 antibodies and your aHUS is stable, this study wants to see what happens when you switch to a new medicine called iptacopan. The main goals are to check if iptacopan is safe and how well it works. This is a "Phase 3" study, which means it's one of the final steps before a new medicine might become widely available. Participants will be monitored closely over a two-year period to see how their aHUS responds to the new treatment.
At a glance
What is this study about?
This study focuses on a condition called atypical Hemolytic Uremic Syndrome, or aHUS for short. aHUS is a very rare and serious kidney disease that can lead to significant health problems. Currently, many people with aHUS are treated with medications called anti-C5 antibodies, which help control the disease.
Researchers are now investigating a new medicine called iptacopan. The purpose of this study is to see what happens when adult patients who are currently stable on their anti-C5 antibody treatment switch to iptacopan. They want to find out if iptacopan is a safe and effective alternative or a new option for managing aHUS. This kind of research is really important because it helps scientists understand new medicines and improve treatment options for people with rare diseases.
This is a Phase 3 study, which means it's a key step in evaluating a new treatment before it can be considered for wider use. By participating, you could help doctors learn more about aHUS and potentially contribute to better treatments for others in the future.
Key takeaways
- This study evaluates switching aHUS treatment from anti-C5 antibodies to iptacopan.
- It's for adults (18+) with stable aHUS who are already on existing treatment.
- The study aims to check if iptacopan is safe and effective.
- Participation involves a screening period, then up to two years on iptacopan.
- Regular health checks and blood tests will be part of the study.
- You can stop participating at any time.
Who may be eligible?
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.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have a diagnosis of aHUS (and no other similar conditions)?
- Have you been on anti-C5 antibody treatment for at least three months, and is it working well for you?
- Are your blood tests (like platelet count and LDH) currently normal, and is your kidney function stable?
- Have you *not* had an aHUS relapse while on your current medication?
- Do you have all your required vaccinations up to date?
What does participation involve?
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.
Potential risks and benefits
Locations (31)
- Novartis Investigative SiteVerified postcodeNanjing, China· Withdrawn
- Novartis Investigative SiteVerified postcodeBeijing, China· Active not recruiting
- Novartis Investigative SiteVerified postcodeBeijing, China· Active not recruiting
- Novartis Investigative SiteVerified postcodeShanghai, China· Withdrawn
- Novartis Investigative SiteVerified postcodeBordeaux, France· Recruiting
- Novartis Investigative SiteVerified postcodeParis, France· Recruiting
- Novartis Investigative SiteVerified postcodeParis, France· Recruiting
- Novartis Investigative SiteVerified postcodeRouen, France· Recruiting
- Novartis Investigative SiteVerified postcodeToulouse, France· Recruiting
- Novartis Investigative SiteVerified postcodeTours, France· Recruiting
- Novartis Investigative SiteVerified postcodeEssen, Germany· Recruiting
- Novartis Investigative SiteVerified postcodeKiel, Germany· Recruiting
Common questions
What is aHUS?
aHUS (atypical Haemolytic Uremic Syndrome) is a rare disease that causes damage to small blood vessels, especially in the kidneys, which can lead to kidney failure.
What is iptacopan?
Iptacopan is a new medication being tested. It works differently from other aHUS treatments and aims to help control the disease.
Will I stop my current aHUS medication?
Yes, if you join this study, you will switch from your anti-C5 antibody treatment to iptacopan after the initial screening period.
How long will the study last?
The main part of the study (called the Core Treatment period) lasts for 12 months, and there's an option to continue for another 12 months in an Extension period.
Will I get good medical care during the study?
Yes, you will receive close medical attention and monitoring from the study doctors and nurses throughout your participation.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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