A phase 3b open-label, multicenter study evaluating physical activity and joint health in previously treated patients ≥12 years of age with severe haemophilia A treated with intravenous recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; efanesoctocog alfa) for 24 months
This study is for people aged 12 and older with severe haemophilia A who have already received treatment. It's testing a new medicine called efanesoctocog alfa (pronounced: eh-fan-eh-sock-toh-kog AL-fa), which is given into a vein. The main goal is to find out if this medicine helps people become more physically active and improves their joint health over a period of 24 months. Researchers will measure physical activity using questionnaires and wearable trackers, and they'll check joint health with special scans and by looking at how joints are feeling. They will also keep an eye on safety, such as any side effects, and how many bleeds participants experience. This is a vital next step to understand how this new treatment might help people with haemophilia A live fuller, more active lives.
At a glance
What is this study about?
Imagine a new type of treatment that could help people with severe haemophilia A live more active lives and protect their joints. That's what this important study is all about. Haemophilia A is a condition where your blood doesn't clot properly, leading to bleeding, especially into joints, which can cause pain and long-term damage. This study is testing a promising new medicine called efanesoctocog alfa, which is given into a vein.
The main aim of the study is to see how this new medicine affects how much physical activity people can do and how healthy their joints become over a 24-month period. Researchers will be using special questionnaires and wearable tracking devices to measure activity levels. They will also use imaging scans, like MRI, to get a detailed look at joint health. They'll be carefully watching for any bleeds, pain, or injuries, and how these might impact daily life, like going to school or work.
Ultimately, this study hopes to show how effective efanesoctocog alfa is in reducing bleeding episodes and improving the quality of life for people with severe haemophilia A. It's a crucial step in understanding if this treatment can help people manage their condition better and enjoy a more active lifestyle with healthier joints.
Key takeaways
- This study is testing a new haemophilia A treatment called efanesoctocog alfa.
- It aims to see if the medicine improves physical activity and joint health.
- Participants will be aged 12 and over with severe haemophilia A.
- The study lasts 24 months and involves regular checks and activity monitoring.
- It's a Phase 3 study, meaning it's a key stage before potential wider use.
Who may be eligible?
To join this study, people must be at least 12 years old and have severe haemophilia A. They should have already received other treatments for their condition in the past.
This study is open to both males and females. The doctors will also check other health details to make sure the study is a good fit and safe for you.
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 12 years old or older?
- Do you have severe haemophilia A?
- Have you been treated for haemophilia A before?
- Are you able to attend regular clinic visits for two years?
What does participation involve?
If you join this study, you'll receive the study medicine, efanesoctocog alfa, which is given into a vein. You will have regular visits to the clinic over a period of 24 months. During these visits, the study team will carry out health checks, take blood samples, and ask you about your symptoms and how you're feeling. You'll also complete questionnaires about your physical activity and wear a tracker to measure how active you are. Joint health will be monitored through examinations and imaging scans, like MRI. There will also be a follow-up period after the treatment ends to ensure your safety.
Potential risks and benefits
Locations (14)
- —UnverifiedCroatia
- —UnverifiedNorway
- —UnverifiedItaly
- —UnverifiedCzechia
- —UnverifiedFrance
- —UnverifiedSweden
- —UnverifiedSpain
- —UnverifiedAustria
- —UnverifiedBelgium
- —UnverifiedGreece
- —UnverifiedSlovenia
- —UnverifiedIreland
Common questions
What is haemophilia A?
Haemophilia A is a condition where your blood doesn't clot properly because you are missing an important protein. This can lead to bleeding inside the body, especially into joints.
What is efanesoctocog alfa?
Efanesoctocog alfa is the name of the new medicine being tested in this study. It's given into a vein to help with blood clotting in people with haemophilia A.
How long will the study last?
The main treatment part of the study will last for 24 months (two years), with some follow-up after that.
Will I know if I'm getting the active medicine?
Yes, in this particular study, called an 'open-label' study, all participants will receive the study medicine, efanesoctocog alfa.
My child is 12, can they join?
Yes, the study is for people aged 12 years and older, but a doctor will need to check if it's suitable for your child based on other health details.
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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