Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
This study aims to understand how the body's immune system responds in people with severe haemophilia A who are treated with a medication called emicizumab. Haemophilia A is a bleeding disorder where the blood doesn't clot properly. Sometimes, the body can develop antibodies (also called inhibitors) that make treatments less effective. This research will look at changes in these antibodies over time. We will do this by using blood samples that are already taken during your routine hospital visits, so you won't need any extra appointments or procedures just for this study. It's a way to learn more about how emicizumab works and how it affects your body's immune response to Factor VIII, which is important for blood clotting.
At a glance
What is this study about?
This research is looking into how a particular medicine, emicizumab, affects people with severe haemophilia A. Haemophilia A is a condition where your blood doesn't clot as it should due to a missing or faulty protein called Factor VIII. Sometimes, especially after treatment, the body can see Factor VIII as a foreign invader and create special proteins called antibodies (or inhibitors) to fight against it. When this happens, it can make treatments for haemophilia less effective.
Emicizumab is a different type of medicine for haemophilia A. This study wants to understand if and how the body's antibodies against Factor VIII change when someone is taking emicizumab. By studying these changes, researchers hope to get a clearer picture of how emicizumab works within the body and how it interacts with the immune system.
The main goal is to gain more knowledge that could help improve care for people with severe haemophilia A in the future. This study won't involve any new treatments or extra appointments for you. It's about gathering information from tests you already have as part of your regular care.
Key takeaways
- Study looks at how emicizumab affects antibodies in severe haemophilia A.
- Uses leftover blood from your routine appointments, no extra tests.
- No changes to your current treatment or medical care.
- Aims to improve understanding of haemophilia A and emicizumab.
- Open to all ages, men and women, with severe haemophilia A treated with emicizumab.
Who may be eligible?
To be part of this study, you need to have been born with severe haemophilia A. You also must be currently receiving treatment with emicizumab. We'll ask for your permission (informed consent) to use your information and leftover samples.
You cannot join the study if you are not taking emicizumab. Also, if you are on medicines that weaken your immune system (immunosuppressive therapy) or if you have HIV with a very low specific immune cell count (CD4 cells less than 200/µl), you won't be able to take part.
This study is open to both men and women of all ages, as long as they meet the criteria above.
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.
- Do I have severe haemophilia A that I was born with?
- Am I currently being treated with emicizumab?
- Am I NOT taking medicines that suppress my immune system?
- Do I NOT have HIV infection with a very low CD4 cell count (less than 200/µl)?
What does participation involve?
If you decide to take part in this study, you won't need to do anything extra. There are no additional visits to the hospital, no new tests, and no extra medicines to take. You will simply continue with your usual appointments and treatment for haemophilia A as planned by your medical team.
The researchers will use very small amounts of leftover blood samples from the routine blood tests you already have during your regular hospital visits. These samples would otherwise be discarded. They will also look at information from your medical records related to your haemophilia care. The study doesn't involve any follow-up that is separate from your standard clinic care. The total duration of your participation will depend on how long you continue on emicizumab treatment and how long the researchers choose to collect samples, but it will not impact your routine medical care.
Potential risks and benefits
Locations (1)
- University Hospital Frankfurt, Goethe UniversityVerified postcodeFrankfurt am Main, Germany· Recruiting
Common questions
What is severe haemophilia A?
It's a serious bleeding disorder where your blood doesn't clot properly because you have very little or no Factor VIII, a protein needed for clotting.
What is emicizumab?
It's a medicine used to help prevent bleeding in people with haemophilia A, by helping the blood clotting process.
What are antibodies or inhibitors?
They are part of your immune system. Sometimes, with haemophilia, the body can produce antibodies against Factor VIII, which can make treatments less effective.
Will I have to take any new medicines or have extra hospital visits?
No, if you join this study, you will continue with your usual treatment and appointments. No extra tests or visits are needed.
What will researchers do with my blood samples?
They will use very small amounts of leftover blood from your routine tests to study the antibodies related to your haemophilia A and emicizumab treatment.
How to find out more
Stephan Schultze-Strasser, Dr.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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