RecruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

This study is investigating a new treatment option, a medicine called Sirolimus, for people with complex vascular malformations. These are unusual growths of blood vessels that can cause pain and other problems, and haven't gotten better with usual treatments like surgery. Researchers want to understand exactly how effective and safe Sirolimus is. They believe this medicine might work by targeting a specific pathway in the body involved in how these malformations develop. The study will involve around 250 children and adults across several hospitals in Europe to gather enough information. If successful, it could offer a new way to help those who haven't had good results from current treatments.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Enrolment target

250

Start

25 Jan 2016

Estimated completion

01 Apr 2030

Vascular Malformations

What is this study about?

Imagine your body's blood vessels are like a network of roads. Sometimes, these roads don't form correctly, creating tangled or unusual pathways. These are called vascular malformations, and they can cause pain, swelling, and other health issues. For many people, existing treatments like surgery or special injections can help. However, for some, these methods don't work well or come with a lot of complications, meaning they're still looking for effective solutions.

This study is exploring a new medicine called Sirolimus (sometimes called Rapamycin) for these difficult-to-treat vascular malformations. Scientists have learned that a specific 'pathway' inside our cells, a bit like a tiny internal signal system, plays a big role in how these malformations grow. Sirolimus works by blocking part of this pathway, which they hope will help to shrink or improve the malformations.

The main goal is to find out if Sirolimus is safe and if it effectively helps improve symptoms for both children and adults who haven't had success with standard treatments. The researchers hope this study will provide clear information about this medicine, potentially offering a valuable new option for patients who currently have limited choices.

Key takeaways

Targets complex vascular malformations that resist standard care.
Investigates the drug Sirolimus (Rapamycin).
Aims to assess both effectiveness and safety.
Includes both children and adults (ages 3 months to 70 years).
Study is in 'Phase III', a later stage clinical trial.
Offers a potential new treatment option for a challenging condition.

Who may be eligible?

To join this study, you would typically need to have complex vascular malformations that haven't improved with regular treatments like surgery or other procedures, or where those treatments caused serious problems. It's important that your blood tests show your body is working well, including your blood cell counts, liver function, and kidney function, as these are important for your safety during the study.

There are also some things that would mean you couldn't join. For example, if you have serious heart problems, a weakened immune system, or severe uncontrolled medical conditions that might make it unsafe for you to take part or affect the study's results. Women who could become pregnant would need to use reliable contraception throughout the study.

Your doctor will look at all your medical information carefully to see if this study is suitable for you. They will explain everything in detail if you are a potential fit.

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 you have a complex vascular malformation that hasn't responded to previous treatments like surgery or injections?
Are you between 3 months and 70 years old?
Are your blood counts (like red blood cells, white blood cells, and platelets) generally healthy?
Is your liver and kidney function within acceptable limits?
Do you have any serious heart conditions or other severe uncontrolled medical conditions?
If you are a woman of childbearing age, are you able to use reliable contraception throughout the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be given the medication Sirolimus. The study will involve regular visits to the hospital for checks and tests. These tests will include blood samples to monitor how your body is responding to the treatment and to make sure it's safe. You'll also have assessments to see how your vascular malformation is changing and if your symptoms are improving. This is a "Phase III" study, meaning it's a larger study designed to confirm the effectiveness and safety of the treatment. The total duration of your participation, including regular follow-up, will be explained by the study team, but typically these kinds of studies can last for several months or longer.

Potential risks and benefits

Taking part in this study could offer the chance to try a new treatment for your vascular malformation if other options haven't worked for you. However, as with any medication, Sirolimus can have side effects. These will be fully explained by the study team. There might also be risks associated with the tests and procedures involved. It's very important to remember that you can stop participating in the study at any time, even if you’ve already started, without it affecting your usual medical care.

Locations (3)

Cliniques Universitaires Saint-Luc, Université Catholique de Louvain Bruxelles
Verified postcode
Brussels, Belgium· Recruiting
CHU Caen
Approximate
Caen, France· Recruiting
Universitätsklinikum Freiburg
Verified postcode
Freiburg im Breisgau, Germany· Not yet recruiting

Common questions

What are vascular malformations?

They are unusual formations of blood vessels that can cause symptoms like pain or swelling. They are different from birthmarks.

What is Sirolimus?

It's a medicine being tested that targets a specific pathway in the body involved in how vascular malformations grow, aiming to improve them.

Who can join this study?

Children and adults (aged 3 months to 70 years) with complex vascular malformations who haven't been helped by usual treatments may be eligible.

Is this medicine available now?

No, it's currently being studied to see if it's safe and effective enough to become a standard treatment for this condition.

What if I change my mind after joining?

You are free to leave the study at any time, for any reason, without it affecting your future medical care.

How to find out more

Laurence M. Boon, MD, PhD

laurence.boon@uclouvain.be + 32-2-764 8020 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.