A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
This study, called MO42623, is for people aged 13 to 69 who have moderate or severe haemophilia A but do not have certain antibodies (Factor VIII inhibitors). It's designed to understand how a specific treatment, emicizumab, impacts their general health, how active they can be, and the health of their joints. Participants will already be on a different haemophilia treatment and will start taking emicizumab as part of the study. This research aims to provide a clearer picture of living with haemophilia A while on this particular medication.
At a glance
What is this study about?
This study is about understanding the real-world effects of a medication called emicizumab for people living with haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly, leading to bleeding problems. There are different types, and this study focuses on moderate or severe forms.
The researchers want to see how emicizumab affects several important aspects of life. They'll be looking at your overall health, how much physical activity you can do, and how your joints are fairing. Joints can sometimes be a problem area for people with haemophilia due to bleeding. By joining this study, you'd be helping doctors learn more about how to best support people with haemophilia A.
It's important to know that this is a 'Phase IV' study. This means the medicine emicizumab is already approved and available. This type of study helps doctors gather more information about how the treatment works in a wider group of people in their everyday lives, beyond the initial trials.
Key takeaways
- This study evaluates emicizumab for moderate or severe haemophilia A.
- It focuses on health, activity, and joint outcomes.
- Participants must not have Factor VIII inhibitors and be aged 13-69.
- You'll switch from your current treatment to emicizumab within the study.
- It aims to provide more real-world information on the treatment.
- Your safety and wellbeing will be carefully monitored throughout.
Who may be eligible?
To be considered for this study, you need to have been diagnosed with either severe or moderate haemophilia A. An important point is that you must not have certain antibodies called 'Factor VIII inhibitors', and you shouldn't have developed them in the last five years. You also need to have been on another regular haemophilia treatment (Factor VIII prophylaxis) for at least 24 weeks before the study starts.
There are also some health checks to make sure the study is a good fit for you, such as having healthy blood, liver, and kidney function. If you are a woman who could become pregnant, you would need to agree to use effective contraception or avoid sexual intercourse during the study and for 24 weeks afterwards.
You wouldn't be able to join if you have another bleeding disorder, have taken emicizumab before, or are planning to have certain joint procedures. Also, if you've had a joint procedure recently (within 2 years) or if it was more than 3 years ago but you're still experiencing pain in that joint, you might not be suitable.
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 moderate or severe haemophilia A?
- Do I currently NOT have Factor VIII inhibitors?
- Have I been on regular Factor VIII treatment for at least six months?
- Am I between 13 and 69 years old?
- Have I NOT taken emicizumab before?
What does participation involve?
If you decide to take part, you will switch from your current haemophilia treatment to emicizumab. The study involves regular visits to the clinic where doctors and nurses will check your overall health, ask about your activity levels, and assess your joint health. You might have blood tests and physical examinations to help track how you're doing on emicizumab. The total duration of your participation in this study would be decided, but for a Phase IV study like this, it generally involves ongoing monitoring and possibly collecting data over a longer period to understand the long-term effects. The research team will explain the full schedule of visits and assessments.
Potential risks and benefits
Locations (28)
- Orthopaedic Institute for ChildrenVerified postcodeLos Angeles, United States
- University of Miami Medical CenterVerified postcodeMiami, United States
- Oklahoma Children's Hospital ? Jimmy Everest CenterVerified postcodeOklahoma City, United States
- Hospital das Clinicas - UNICAMPVerified postcodeCampinas, Brazil
- Hospital das Clínicas Faculdades Médicas de Ribeirão PretoVerified postcodeRibeirão Preto, Brazil
- Hamilton Health Sciences CorporationVerified postcodeHamilton, Canada
- Charité Universitätsklinikum BerlinVerified postcodeBerlin, Germany
- Universitätsklinikum BonnVerified postcodeBonn, Germany
- Észak-Pesti Centrumkórház - HonvédkórházVerified postcodeBudapest, Hungary
- AOU Federico IIVerified postcodeNaples, Italy
- Policlinico Univ. A. GemelliVerified postcodeRome, Italy
- IRCCS Ca' Granda Ospedale Maggiore PoliclinicoVerified postcodeMilan, Italy
Common questions
What is haemophilia A?
Haemophilia A is a condition where your blood doesn't clot properly, which can lead to bleeding problems, especially in your joints and muscles.
What is emicizumab?
Emicizumab is a medication used to help prevent bleeding in people with haemophilia A. It works differently from other common haemophilia treatments.
What are Factor VIII inhibitors?
Factor VIII inhibitors are antibodies that your body can sometimes make. They can stop common haemophilia treatments from working properly.
Will I stop my current haemophilia treatment if I join?
Yes, if you join this study, you will switch from your current Factor VIII prophylaxis to emicizumab as your treatment.
How long will the study last?
The study aims to gather ongoing information. The study team will explain the expected duration of your individual participation and the schedule of visits.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.