A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study is gathering information on Refixia/REBINYN, a treatment for males with Haemophilia B. We want to understand how it works and if there are any side effects when used over several years. If you join, you'll continue to receive your usual treatment as decided by your doctor. You'll have regular clinic visits just as you do now, where your doctor might check your health and ask about any side effects. You'll also be asked to keep track of any bleeds and your treatment, and to fill out questionnaires about your quality of life and physical activity. Your participation could last for 4 to 9 years, but you are free to leave the study at any time without affecting your medical care.
At a glance
What is this study about?
This research study is for males who have Haemophilia B and are being treated with a medicine called Refixia/REBINYN. Haemophilia B is a rare inherited disorder where your blood doesn't clot properly due to a lack of a specific protein called Factor IX. This can lead to longer bleeding after injuries.
The main aim of this study is to collect information on how well Refixia/REBINYN works to prevent bleeds and whether it causes any side effects when used regularly over a long period. We already know this treatment helps, but understanding its long-term effects is very important for patients' health and well-being. This information helps doctors understand the best ways to care for people with Haemophilia B.
By taking part, you'll be contributing to valuable knowledge that could help many other people living with Haemophilia B in the future. The study is designed to fit in with your normal medical care, so it shouldn't feel too different from your usual check-ups.
Key takeaways
- This study collects long-term information on a Haemophilia B treatment called Refixia/REBINYN.
- It aims to understand how well the medicine works and if there are any side effects over time.
- Only males with Haemophilia B already using or starting Refixia/REBINYN can join.
- You'll continue your usual treatment and have regular check-ups.
- You'll track bleeds and fill out questionnaires about your quality of life.
- Participation lasts 4-9 years, with the option to withdraw at any time.
Who may be eligible?
This study is specifically for males of any age who have Haemophilia B and who are already using or are about to start using Refixia/REBINYN as a regular treatment to prevent bleeds. Importantly, the decision to start or continue this particular medicine must have been made by you (or your parent/guardian if you're under 18) and your doctor, completely separate from whether you join this study.
This means if your doctor has already decided that Refixia/REBINYN is the best treatment for you, then you might be able to join. You'll need to be able to understand what the study involves and agree to take part by signing a consent form.
You won't be able to join if you've already taken part in this specific study before. Also, if you're known to be allergic to Refixia/REBINYN or similar medicines, or if your body has developed a problem where it makes antibodies against Factor IX (called an "inhibitor"), you wouldn't be able to participate. If you have difficulties understanding or cooperating due to mental health issues, or a language barrier that can't be overcome, you wouldn't be eligible either.
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 male?
- Do you have Haemophilia B?
- Are you currently using or about to start Refixia/REBINYN as regular treatment?
- Has your doctor already decided this treatment is right for you, separately from the study?
- Have you not previously taken part in this specific study?
- Are you able to understand the study information and agree to take part?
What does participation involve?
If you decide to take part in this study, you'll continue with your regular treatment of Refixia/REBINYN, just as prescribed by your study doctor. Your clinic visits will be much like your usual appointments. During these visits, your doctor might do some routine tests if they think it's helpful.
They will also ask if you've had any side effects since your last visit. We'll ask you to keep a record of any bleeds you have, how you treat them, and when you take your regular Refixia/REBINYN dose. You'll also be asked to fill out questionnaires about how your Haemophilia B affects your daily life and your ability to be physically active.
Your participation in this study could last anywhere from 4 to 9 years, depending on when you start. Remember, you can choose to leave the study at any time, for any reason, without it affecting your medical care.
Potential risks and benefits
Locations (29)
- AKH - Klin. Abt. f. Haematologie u. HaemostaseologieVerified postcodeVienna, Austria
- Cliniques universitaires Saint-Luc - Service HématologieVerified postcodeBrussels, Belgium
- UZ Antwerpen - UZA - Kinderhemato-OncologieVerified postcodeEdegem, Belgium
- UZ Leuven - Hart en VaatziektenVerified postcodeLeuven, Belgium
- University of Calgary Cumming School of MedicineVerified postcodeCalgary, Canada
- Univ of Alberta Hospital ResVerified postcodeEdmonton, Canada
- CancerCare ManitobaVerified postcodeWinnipeg, Canada
- Health Science CentreVerified postcodeSt. John's, Canada
- Hamilton Health Sciences Corp, OntarioVerified postcodeHamilton, Canada
- Hamltn Hth Sci/McMstr Child HospVerified postcodeHamilton, Canada
- The Hospital for Sick ChildrenVerified postcodeToronto, Canada
- KBC Zagreb, Rebro, Hemofilija centarVerified postcodeZagreb, Croatia
Common questions
What is Haemophilia B?
Haemophilia B is a health condition where your blood doesn't clot properly because you are missing a protein called Factor IX, leading to more bleeding.
What is Refixia/REBINYN?
Refixia/REBINYN is a medicine used to treat Haemophilia B by replacing the missing Factor IX, helping your blood clot better and preventing bleeds.
Will I get a different treatment in this study?
No, you will keep receiving the same Refixia/REBINYN treatment that your own doctor has already decided is best for you.
How long will I be in the study?
You could be in the study for 4 to 9 years, depending on when you start, but you can leave at any time.
Can I stop being in the study?
Yes, you can leave the study at any time for any reason, and it won't affect your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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