A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
This study is for adults with a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. Many people with PNH are treated with medicines that block something called C5, which helps their red blood cells. If you're on one of these treatments and your blood count (haemoglobin) is stable, this study wants to see if switching to a new daily tablet called iptacopan is just as good, or even better. Researchers will measure blood levels to see if iptacopan keeps your haemoglobin healthy. They'll also check if the new medicine is safe and doesn't cause too many side effects. This is a big study (Phase III) to confirm if iptacopan could be a good treatment option for PNH.
At a glance
What is this study about?
This study is about a health problem called Paroxysmal Nocturnal Hemoglobinuria, or PNH. PNH is a rare condition where your red blood cells get attacked and destroyed by your own immune system, which can make you feel very tired and unwell. Currently, many people with PNH are treated with special medicines that help stop this attack on their red blood cells. These medicines often work by blocking something in your body called C5, and they can make a big difference, helping your blood count (haemoglobin) get to a healthy level.
Now, scientists are testing a new medicine called iptacopan. This medicine is different because it's a tablet you take twice a day, and it works in a slightly different way to protect your red blood cells. This particular study is designed for adults with PNH who are already being successfully treated with a C5-blocking medicine and have a good blood count. The main goal is to see if switching from their current treatment to iptacopan is just as effective at keeping their blood healthy and if it's safe to use.
This is an important step in research because if iptacopan works well and is safe, it could offer a new, potentially more convenient, option for people living with PNH. The study is called a 'Phase III' trial, which means it's one of the final stages of testing before a new medicine might become widely available.
Key takeaways
- Tests a new daily tablet (iptacopan) for PNH.
- For adults with PNH already stable on C5-blocking treatments.
- Checks if iptacopan is safe and works just as well.
- Aims to find new and potentially more convenient treatment options.
- This is a Phase III study, close to widespread use if successful.
Who may be eligible?
This study is looking for adult volunteers who have a specific rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH.
To be considered, you must already be receiving treatment for your PNH with a medicine that blocks something called C5. Importantly, your current treatment needs to be working well, meaning your blood count (haemoglobin level) should be stable and at a certain healthy level.
Both men and women are welcome to take part, as long as they are 18 years old or older. The research team will review everyone's medical history to make sure the study is a good fit and safe for them.
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 Paroxysmal Nocturnal Hemoglobinuria (PNH)?
- Are you currently being treated for PNH with a C5-blocking medicine?
- Is your current PNH treatment working well, with a haemoglobin level of 10 g/dL or higher?
What does participation involve?
If you decide to join this study, you will switch from your current PNH medication to the new study medicine, iptacopan. This is a tablet you will take twice a day. The study will involve regular visits to the clinic where you will have blood tests. These tests will check your blood count (haemoglobin levels) to see how well the new medicine is working, and also to monitor your general health and check for any side effects. You will have blood tests before starting iptacopan, and then during the study at specific times, including around Day 126 and Day 168. The study team will guide you through all the steps, from starting the new medicine to following up on your progress.
Potential risks and benefits
Locations (4)
- —UnverifiedSpain
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedItaly
Common questions
What is PNH?
PNH is a rare blood condition where red blood cells are destroyed too early by the body's immune system, which can cause anaemia and other related health issues.
What is iptacopan?
Iptacopan is a new medicine being tested. It's a tablet taken twice a day, designed to help protect red blood cells in people with PNH.
Why is this study important for PNH patients?
This study aims to see if iptacopan could be a good, potentially more convenient, treatment option for adults with PNH, especially for those already stable on other medications.
Will I have to stop my current PNH treatment?
Yes, if you join the study, you would switch from your current PNH C5-blocking medication to take iptacopan instead.
How long does the study last?
The study mentions checking blood levels at Day 126 and Day 168, indicating it will last for several months, with ongoing monitoring.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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