AuthorisedTherapeutic exploratory (Phase II)Interventional

MAV-RAPA: Prospective evaluation of the efficacy of sirolimus (Rapamune) in the treatment of superficial arteriovenous malformations.

This study, called MAV-RAPA, is investigating a medicine called Rapamune for adults who have a condition where blood vessels grow abnormally near the skin, known as superficial arteriovenous malformations (AVMs). The main goal is to see if Rapamune can shrink these growths by at least 30% within the first year of treatment, using special scans (angiograms) to measure them. This is a Phase II study, meaning researchers are exploring how effective the treatment is. They will also check how well the treatment is working at three, six, and nine months. They hope this medicine can offer a new way to help manage AVMs.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Centre Hospitalier Universitaire Amiens Picardie

Enrolment target

Start

17 Jan 2025

superficial arteriovenous malformations

What is this study about?

You might be reading this because you, or someone you care about, has a condition called a superficial arteriovenous malformation (AVM). This is when blood vessels grow abnormally, often appearing on or just under the skin. The MAV-RAPA study is looking into a medication called Rapamune (which is also known by its scientific name, sirolimus) as a possible treatment for these AVMs.

The main aim of this study is to find out how effective Rapamune is at shrinking these unusual blood vessel growths. Researchers will be giving adults with AVMs this medication and then regularly checking, using special types of scans, to see if the AVMs reduce in size by at least 30% during the first year. They will also be looking closely at how well the treatment performs at different points: after three, six, and nine months.

This is a 'Phase II' study, which means it's an important stage where scientists are further exploring if a new treatment works and is safe. If the results are positive, it could mean a new option for people living with superficial AVMs, potentially helping to manage the condition and improve quality of life.

Key takeaways

This study is testing a drug called Rapamune for adults with certain abnormal blood vessel growths (AVMs).
The main goal is to see if the drug can shrink AVMs by at least 30% within a year.
It's a 'Phase II' study, which means researchers are exploring how well the treatment works.
Regular scans and check-ups will be part of the study.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you need to be an adult, 18 years old or older. The study is open to both men and women.

Because this study focuses on specific types of blood vessel growths, it's important that your condition is a superficial arteriovenous malformation. Your doctor would help determine if your specific condition matches what the study is looking for.

There might be other health conditions or medications that would mean you couldn't take part. These details would be discussed fully with the study doctor to make sure the study is safe and right 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 'superficial arteriovenous malformation'?
Have you discussed your willingness and ability to attend regular appointments and scans?
Are you comfortable with taking a new study medication?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be taking medication called Rapamune. The study also mentions two strengths of another medicine, Solupred, though how these fit into the treatment plan would be fully explained by the study team.

Throughout the study, you would have regular appointments to see how you're responding to the treatment. These appointments would include special scans (angiograms) to measure your AVM. These scans are important for checking if the AVM is shrinking. You would have these assessments at different times, including at three, six, nine months, and at the end of the first year to check the main goal of the study.

The study would involve regular check-ups with the medical team to monitor your health and how you're reacting to the medication. The total time for your active involvement in the study's main evaluations would be one year.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. A possible benefit of this study is that Rapamune could help shrink your AVM, potentially reducing symptoms and improving your health. However, like all medicines, Rapamune can have side effects, which the study team would explain in detail. You would be closely monitored for any reactions. It's very important to understand that your participation is completely voluntary; you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is an arteriovenous malformation (AVM)?

It's an abnormal tangle of blood vessels that connects arteries and veins, often found in or just under the skin.

What is Rapamune?

Rapamune is a medicine, also known as sirolimus, that is being studied to see if it can help treat AVMs.

How long will this study last for me?

The main part of the study where your AVM is measured and reviewed will last for one year.

Will I have to have scans?

Yes, you will have special scans, called angiograms, to measure your AVM and see if it's shrinking.

Can I stop participating in the study at any time?

Yes, you are free to leave the study at any point, and it won't affect your ongoing medical care.