A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This four-year study aims to understand how Elocta, a medicine for haemophilia A, helps protect joints over a long period. Haemophilia A can cause frequent bleeding into joints, leading to pain and long-term damage. Elocta is designed to stay in the body longer than other treatments, which might offer better protection against these bleeds and help maintain healthier joints. This study will observe people who are already using Elocta as part of their regular treatment to see how well it works in real-life situations. Researchers will gather information to see if Elocta can reduce joint problems and improve the quality of life for those with haemophilia A. It's an important step in finding the best ways to manage this condition.
At a glance
What is this study about?
This study is looking at how a medicine called Elocta helps people with haemophilia A keep their joints healthy over time. Haemophilia A is a condition where the blood doesn't clot properly, which can lead to bleeding, especially into joints like elbows, knees, and ankles. These bleeds can be painful and, over many years, can cause lasting damage, making it hard to move your joints.
Doctors often recommend early treatment with medicines that replace the missing clotting factor (Factor VIII) to prevent these bleeds and protect joints. Although these treatments work well, some people still experience joint bleeds. Elocta is a newer type of Factor VIII medicine that stays in the body for longer. This means it might offer more consistent protection against bleeds and give doctors more flexibility in how they treat individual patients.
The study will last for four years and will observe people who are already using Elocta as part of their regular care. Researchers want to see if Elocta helps keep joints healthy in the long run. They will collect information on how patients are doing, how often they have bleeds, and how their joints are holding up. This information will help doctors understand the best ways to use Elocta to prevent joint damage for people with haemophilia A.
Key takeaways
- This study observes the long-term effects of Elocta on joint health in people with haemophilia A.
- It will run for four years (48 months).
- Participants will continue their current Elocta treatment as usual; no new medications are given.
- The aim is to understand how Elocta helps prevent joint damage in real-life situations.
- The findings could improve care for everyone with haemophilia A.
- You can withdraw at any time.
Who may be eligible?
To join this study, you need to have haemophilia A and already be taking Elocta medicine to prevent bleeds. If you are under 18, a parent or legal guardian will need to give their permission for you to take part, and you will also be asked if you understand and agree.
It's important that you are not currently in another study where you are trying out a new, experimental medicine. Also, you cannot have developed certain antibodies (proteins) in your blood that would stop Factor VIII treatments like Elocta from working properly.
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 you have a diagnosis of haemophilia A?
- Are you currently receiving Elocta as preventative treatment?
- Are you not currently involved in another clinical study testing new drugs?
- Have you not had a recent test showing you have Factor VIII antibodies (inhibitors) at a high level?
What does participation involve?
If you take part in this study, you will be observed over a period of four years. Because this is an 'observational' study, it means you will continue your regular Elocta treatment as your doctor has prescribed, just as you normally would. You won't be given new or different medicines specifically for the study. Researchers will collect information about your health, such as how often you have bleeds, how your joints are feeling, and potentially use information from mobile apps or wearable devices if you use them to track your health. You will have regular check-ups as part of your usual care, and the study will gather information during these visits. The total duration of your participation will be 48 months, or four years.
Potential risks and benefits
Locations (53)
- University Hospital BrnoVerified postcodeBrno, Czechia
- University Hospital OstravaVerified postcodeOstrava, Czechia
- Dept. of Pediatric Haematology and Oncology, University Hospital MotolVerified postcodePrague, Czechia
- Lastehaigla, Tallinn (Tallinn Children´s Hospital)Verified postcodeTallinn, Estonia
- The North Estonia Medical Centre Hematoloogiakeskus, RegionalhaiglaVerified postcodeTallinn, Estonia
- Helsinki University Central Hospital, New Children HospitalVerified postcodeHelsinki, Finland
- Turku University Central Hospital, Paediatric and adolescent haematology and oncology clinicVerified postcodeTurku, Finland
- Charité-Universitätsmedizin Berlin Campus Virchow KlinikumVerified postcodeBerlin, Germany
- Universitätsklinikum Bonn AöR, Institut für Experimentelle Hämatologie und TransfusionsmedizinVerified postcodeBonn, Germany
- Hämostaseologie/Hämophiliezentrum, Medizinische Klinik 2 Institut für Transfusionsmedizin, UniversitätsklinikumVerified postcodeFrankfurt, Germany
- Universitätsklinikum Frankfurt - Klinik für Kinder- und JugendmedizinVerified postcodeFrankfurt, Germany
- Universitätsklinikum Hamburg-Eppendorf (UKE)Verified postcodeHamburg, Germany
Common questions
What is haemophilia A?
It's a rare condition where your blood doesn't clot properly, leading to bleeding, especially in your joints.
What is Elocta?
Elocta is a medicine (a type of factor VIII) that helps your blood clot, and it stays in your body longer than some other treatments.
Will I have to take new medicine for this study?
No, you will keep taking Elocta as your own doctor has already prescribed. This study simply observes its effects over time.
How long will the study last?
The study will last for four years (48 months).
Can I stop being part of the study once I've joined?
Yes, you can choose to leave the study at any time, and it won't affect your regular medical care.
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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