An 18-month low-interventional prospective, multicentre study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa
This study, called a Phase IV trial, is for people in the UK living with haemophilia A or B. It aims to understand how effective current treatments, specifically Elcocta and Alprolix, are at keeping their joints healthy over time. Researchers will follow participants for 18 months, regularly checking their joints using ultrasound scans to see if the treatments help prevent joint damage. They will also look at how often bleeding occurs and how patients feel about their physical activity and pain. This information will help doctors understand the long-term benefits of these medicines for daily life and joint health.
At a glance
What is this study about?
This research study is for people in the UK who have haemophilia A or B. Haemophilia is a condition where your blood doesn't clot properly, which can lead to bleeding, especially into joints, causing pain and damage over time. The study wants to find out how well certain medicines, called Elcocta and Alprolix, protect joints in people who take them regularly to prevent bleeding (this is called prophylaxis).
Doctors will follow participants for 18 months, checking their joints closely. They will use special ultrasound scans (called HEAD-US scores) to look at the health of the joint lining (synovium), the cushioning material (cartilage), and the bones. They'll compare these scans from the beginning to the end of the study to see if the treatment has made a difference.
The study also looks at other important things, like how often bleeding happens (especially in particular joints), how much physical activity people can do, and their pain levels. Gathering all this information will help doctors understand the full benefits of these treatments in real-world settings and improve care for people with haemophilia in the future.
Key takeaways
- This study is for adults with haemophilia A or B.
- It aims to see how well Elcocta and Alprolix protect joints over 18 months.
- Joint health will be checked using ultrasound scans.
- You will continue your usual treatment.
- The study also looks at bleeding rates and quality of life.
Who may be eligible?
This study is looking for adults aged 18 and over, both men and women, who have been diagnosed with haemophilia A or B.
Willingness to continue your current treatment with either Elcocta or Alprolix as prescribed is also important, as the study observes how these particular medications affect your joint health over time.
There may be other specific medical details that determine if this study is right for you, so it's always best to discuss it with your doctor.
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 haemophilia A or B?
- Are you already taking Elcocta or Alprolix to prevent bleeding?
- Are you able to attend regular check-ups over 18 months?
What does participation involve?
If you decide to take part, you will continue to receive your prescribed medicine (either Elcocta or Alprolix) as you normally would. Over the 18 months of the study, you'll have regular check-ups. These will include ultrasound scans of your joints to see how they are doing. You'll also be asked to complete questionnaires about your physical activity, how often you experience bleeding, and your pain levels. The study is designed to fit in with your existing treatment plan with minimal extra visits.
Potential risks and benefits
Locations (10)
- —UnverifiedHungary
- —UnverifiedIreland
- —UnverifiedCzechia
- —UnverifiedItaly
- —UnverifiedCroatia
- —UnverifiedSpain
- —UnverifiedRomania
- —UnverifiedFrance
- —UnverifiedSlovenia
- —UnverifiedBulgaria
Common questions
What is haemophilia?
Haemophilia is a condition where your blood doesn't clot properly, leading to longer bleeding. This can cause problems like joint damage over time.
What are Elcocta and Alprolix?
These are medicines used by people with haemophilia to help their blood clot better and prevent bleeding episodes.
Why are joints so important in this study?
Bleeding into joints is common in haemophilia and can cause pain and long-term damage, so the study wants to see how well these medicines protect them.
Will I get a new medicine?
No, this study looks at the medicines you are already taking (Elcocta or Alprolix) and how they work in real life. It's not testing a new drug.
How long will the study last?
The study will follow participants for 18 months.
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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