RAPAMALYMPH - Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
This study, called RAPAMALYMPH, is testing a medicine called Rapamycin (also known as Rapamune) in people with a type of growth in their head or neck called a lymphatic malformation. These growths, sometimes called lymphangiomas, can be difficult to treat and can cause problems. The main goal is to find out if Rapamycin can make these growths smaller. Doctors will use special scans, called MRIs, to measure the size of the growth before treatment and again after three months of taking the medicine. If the growth shrinks by one-fifth or more, the treatment will be considered helpful. We will also check for any side effects and see how the growth changes over a longer period.
At a glance
What is this study about?
This study is called RAPAMALYMPH, and it's looking into a medicine called Rapamycin (which you might also know as Rapamune) for a specific type of health problem. The problem is a rare growth in the head and neck area, called a lymphatic malformation. These are sometimes called lymphangiomas. They are basically collections of abnormal fluid and blood vessels that can cause swelling or other issues and are often hard to treat with usual methods.
The main aim of this study is to see if Rapamycin can help shrink the size of these growths. To do this, doctors will use special scans called MRIs. Imagine taking a detailed picture of the malformation before you start the medicine and then again after three months of treatment. They will compare these pictures to see if the growth has gotten smaller. If it shrinks by at least one-fifth of its original size, that will be considered a positive sign that the medicine is working.
We'll also be keeping a close eye on a few other things. This includes checking for any side effects from the medicine every month. We'll also use other methods, like photographs and videos, to see how the malformation looks and feels, and how it might be affecting areas like your throat. These checks will happen at three, six, and twelve months to understand how the treatment affects the malformation over time and even after you stop taking the medicine. We're also curious about how the medicine works on a tiny, cellular level.
Key takeaways
- This study tests Rapamycin for hard-to-treat lymphatic malformations in the head/neck.
- The main goal is to see if the medicine shrinks the malformation.
- MRI scans will be used to measure any changes in size.
- Side effects will be checked monthly.
- Participation involves taking daily medication and regular visits for up to a year.
- It's for adults 18 years and older with specific types of malformations.
Who may be eligible?
This study is open to both men and women who are 18 years old or older. There isn't an upper age limit mentioned, so if you're an adult living with a lymphatic malformation in your head or neck that's been difficult to treat, this study might be for you.
The important thing is that the lymphatic malformation must be in the cervico-facial area, which means your head and neck region, and it should be considered 'poor prognosis,' meaning it's been particularly challenging to manage with other treatments.
If you fit this general description, you could be eligible. However, there will be more specific criteria that your doctor will need to check to make sure the study is safe and suitable 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 18 years old or older?
- Do you have a lymphatic malformation in your head or neck area?
- Has your lymphatic malformation been difficult to treat with other methods?
- Are you able to take an oral liquid medicine?
What does participation involve?
If you decide to take part in this study, you would be taking a medicine called Rapamune, which is an oral solution (a liquid you drink). You would take this medicine as prescribed by the study doctors. A very important part of the study involves regular MRI scans. You'll have an MRI before you start treatment, and then again three months after the medicine has reached a steady level in your body. Sometimes, if you have an infection or injury, this scan might be delayed by a week or two.
Beyond the main scans, you’ll also have follow-up MRI scans at 6 months and 1 year to see how things are progressing. Doctors will check for any side effects every month. You’ll also have clinical exams where they might take photos or videos of the malformation, especially if it affects your throat. All these assessments help the doctors understand if the medicine is working and if it's safe. The total duration of your participation would involve treatment and follow-up for a year, sometimes even six months after stopping the medicine.
Potential risks and benefits
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Common questions
What is a lymphatic malformation?
It's a rare growth of abnormal fluid-filled vessels, often seen in the head or neck, that can cause swelling and other problems.
What is Rapamycin?
It's a medicine being tested in this study to see if it can help shrink the lymphatic malformations.
How will doctors know if the medicine is working?
They'll use special scans called MRIs to measure the size of the growth before and after treatment. A significant shrinkage will be seen as a positive result.
Are there side effects?
Like all medicines, Rapamycin can have side effects. The study team will check for these every month and tell you about them if you consider joining.
How long does the study last?
Your participation will involve taking the medicine and having follow-up checks for about a year, including assessments after stopping the treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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