Monocentric Pilot Trial evaluating the safety and efficacy of Regorafenib in Arterio-Venous Malformations that are refractory to standard care
This study is a small, early-stage trial looking into a medicine called regorafenib for a condition known as Arterio-Venous Malformations, or AVMs. AVMs are unusual connections between blood vessels. This trial is specifically for adults whose AVMs haven't responded well to the standard treatments. We want to check if regorafenib is safe and if it helps by looking at several things. We'll ask participants about their quality of life and how their pain levels are. We'll also use medical scans like ultrasound, MRI, and special X-rays (arteriography) to see if the AVMs change in size over 12 months. Additionally, we'll monitor if the medicine helps people with their daily activities. This is a "Phase II" study, meaning it's still in the early stages of testing this new use for the medicine.
At a glance
What is this study about?
This study is looking at a medicine called regorafenib as a possible new treatment for a condition known as Arterio-Venous Malformations (AVMs). AVMs are basically abnormal tangles of blood vessels where arteries connect directly to veins, bypassing the tiny capillaries that usually slow blood flow down. This can cause various problems depending on where they are in the body, such as pain or swelling.
Currently, there are standard ways to treat AVMs, but they don't always work for everyone. This study is specifically for adults whose AVMs haven't gotten better with these usual treatments. The main goal is to find out if regorafenib is both safe to use and if it can actually help improve the condition. This particular trial is called a "Therapeutic exploratory (Phase II)" study, which means it's an earlier stage of research where we're trying to understand if the new treatment has a hopeful effect and what the right dose might be if it does.
To see if regorafenib is helping, the researchers will look at a few key things. Patients will be asked to fill out questionnaires about their quality of life, focusing on whether they feel better overall. The study will also pay close attention to any changes in pain levels. In addition to patient reports, doctors will use special medical imaging techniques like ultrasound, MRI scans, and a type of X-ray called arteriography to measure the size of the AVMs and see if they shrink over a 12-month period. They will also look at how the AVM affects daily activities, such as how easy it is to do everyday tasks.
Key takeaways
- This is a study for adults with Arterio-Venous Malformations (AVMs).
- It's for people whose AVMs haven't improved with other treatments.
- The study is testing a medicine called regorafenib.
- Researchers will check if the medicine improves quality of life, pain, and AVM size.
- Participation will involve regular check-ups and scans over at least 12 months.
Who may be eligible?
To take part in this study, you must be an adult, aged 18 years or older. There is no upper age limit, meaning older adults can also participate.
Both men and women are welcome to join this study. The researchers are looking for a diverse group of participants to understand how the medicine works across different people.
Crucially, this study is for people whose Arterio-Venous Malformations (AVMs) have not responded well to other standard treatments. If your AVM has improved with other therapies, this study might not be suitable for you.
- Are you 18 years old or older?
- Do you have an Arterio-Venous Malformation (AVM)?
- Have your AVMs not improved with standard treatments?
- Are you able to attend regular appointments and medical scans over 12 months?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you will be given the medicine regorafenib. Throughout the study, the research team will closely monitor your progress. This will involve filling out questionnaires about your quality of life and pain levels. You will also have regular check-ups and medical scans, such as Doppler ultrasounds, MRI scans, and arteriographies, to see how your AVM is changing in size. These scans will be done over a 12-month period. The study will also assess any difficulties you might have with daily activities. The total duration of your participation, including all follow-up appointments and assessments, will extend for at least 12 months.
Potential risks and benefits
Locations (1)
- —Belgium
Common questions
What is an Arterio-Venous Malformation (AVM)?
An AVM is an unusual tangle of blood vessels where arteries connect directly to veins, rather than through small capillaries, causing abnormal blood flow.
What is regorafenib?
Regorafenib is a medicine being tested to see if it can help treat AVMs, especially when other treatments haven't worked well.
Why is this study happening?
This study aims to find out if regorafenib is a safe and effective treatment for AVMs, especially for people who haven't found help with standard treatments.
What are the main things the study will look at?
The study will check if your quality of life improves, if your pain goes down, and if the size of your AVM changes over 12 months using medical scans.
How long will I be in the study?
Your participation in the study, including all treatments and check-ups, will last for at least 12 months.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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