RecruitingPHASE2INTERVENTIONAL

Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

This research study is investigating an oral medicine called rapamycin for children and teenagers (up to 18 years old) who have lymphatic malformations in their head and neck area. These malformations are unusual growths of lymphatic vessels. The main goal is to see if rapamycin can effectively shrink these malformations. Researchers will measure the size of the growth using MRI scans before and after treatment. They also want to understand if the medicine helps improve a child's overall quality of life and reduces problems like bleeding if the malformation affects the lining of the mouth or throat. The treatment lasts for six months, and the study is in its second phase of testing.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

University Hospital, Lille

Enrolment target

Start

25 Jun 2018

Estimated completion

01 Feb 2027

Lymphatic Malformation Pediatric

What is this study about?

You might be reading this because you, or someone you care for, has a lymphatic malformation, especially one affecting the head and neck. These are growths that happen when the body's lymphatic system (which is part of our immune system) doesn’t develop quite right. They can sometimes cause problems like pain, difficulty breathing or swallowing, or bleeding, depending on where they are and how big they are.

This study, called a clinical trial, is looking into a medicine called rapamycin. It's already used for other health conditions, but researchers want to see if it can help children and teenagers with specific types of lymphatic malformations. The hope is that this medicine might offer a new way to manage these conditions, especially when surgery or other treatments aren't possible or carry too many risks.

For this trial, children will take rapamycin by mouth for six months. The medical team will carefully monitor whether the malformation shrinks, how it affects the child's daily life, and if symptoms like bleeding improve. The results of studies like this help doctors learn more about the best ways to treat these conditions in the future.

Key takeaways

This study explores a new oral medicine (rapamycin) for lymphatic malformations.
It's for children and teenagers with specific head/neck malformations causing problems.
The treatment lasts for six months, with regular checks and MRI scans.
Goals are to shrink the malformation, improve quality of life, and reduce symptoms.
Doctors will carefully check if the child is suitable for the study.

Who may be eligible?

To join this study, children and teenagers up to 18 years old must have a specific type of lymphatic malformation in their head, face, or neck area. This malformation should be causing ongoing pain or making it hard to breathe or swallow. It's important that other treatments, like surgery, aren't suitable or would be too risky.

Children also need to be generally stable and reasonably healthy, with good results from routine blood tests for things like blood count, kidney, and liver function. They shouldn't have any serious infections or other serious illnesses. If they are taking certain other medications, they might not be able to join because of how those medicines could react with rapamycin. Girls who are able to get pregnant must have a negative pregnancy test, and pregnant or breastfeeding individuals cannot take part.

There are also some things that would prevent someone from joining. For example, if someone has very serious kidney or liver problems, issues with digesting medicines, or severe allergies to peanuts or soya (which might be in the medicine). Also, if someone is already taking other immune-suppressing medicines or steroids long-term, they would need to have stopped those for a period before considering this study.

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.

Is the child aged 0-18 years old?
Does the child have a lymphatic malformation in the head/neck causing pain or difficulty breathing/swallowing?
Are other treatments like surgery not a good option for the child?
Does the child have generally healthy blood test results?
Is the child not currently pregnant or breastfeeding (if applicable)?
Is the child not taking certain medications that might react with rapamycin?

Answer every question to see your result.

What does participation involve?

If you or your child joins this study, they would take the medicine rapamycin by mouth every day for six months. Before starting, and at certain times during the study, they would have MRI scans to measure the lymphatic malformation. The medical team would also regularly check how the malformation is affecting daily life and any symptoms like bleeding. There will be regular check-ups and blood tests to monitor health and the effects of the medicine. The full duration of participation, including follow-up after the six months of treatment, would be explained in detail by the study team.

Potential risks and benefits

Taking part in this study could potentially offer a new treatment option for your child's lymphatic malformation, possibly leading to shrinkage of the growth, improved quality of life, and reduced symptoms like bleeding. However, like all medicines, rapamycin can have side effects, and not everyone responds to treatment. The study team will explain all known potential risks and side effects in detail. Remember, joining a study is always voluntary, and you are free to withdraw your child at any time without affecting their future medical care.

Locations (2)

Hôpital Jeanne de Flandres, CHU
Verified postcode
Lille, France· Recruiting
Hu Robert Debre Aphp - Paris
Verified postcode
Paris, France· Recruiting

Common questions

What is a lymphatic malformation?

It's a non-cancerous growth or swelling that happens when tiny tubes called lymphatic vessels don’t form properly, often around the head and neck.

What is rapamycin?

Rapamycin is a medicine that can affect the immune system and cell growth, and this study aims to see if it can help shrink these malformations.

How long will the treatment last?

Children in this study will take the medicine by mouth for a period of six months.

How will we know if the treatment is working?

The team will use MRI scans to measure the size of the malformation and will also ask about how it's affecting daily life and any symptoms.

Can my child join if they are on other medicines?

Some medicines can interfere with rapamycin, so the study team will need a full list of all medications your child is taking to check if they are eligible.

How to find out more

Pierre Fayoux, MD

pierre.fayoux@chru-lille.fr 3 20 44 50 67 Official trial record

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