Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

TOPical sirolimus in linGUal microkystic lymphatic malformation

This research is looking into a new treatment for a rare tongue condition called lingual microcystic lymphatic malformation, or LMLM. This condition causes small cysts on the tongue that can lead to problems with speaking, eating, and breathing, as well as pain and bleeding. The study will test a cream containing a medicine called sirolimus, applied directly to the tongue, for 12 weeks. Doctors will track changes in the severity of the condition using photographs, and patients will share how they feel about their symptoms and overall quality of life. The aim is to see if this cream can help reduce the symptoms and improve daily living for those with LMLM.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Centre Hospitalier Regional Universitaire De Tours

Enrolment target

Start

22 Aug 2024

Lingual microcystic lymphatic malformations (LMLMs) are rare congenital vascular malformations presenting as clusters of cysts filled with lymph fluid or blood. They are responsible for a heavy burden even with small well-limited lesions because of oozing bleeding infections or even speech chewing or breathing impairment. Pain and aesthetic prejudice are also frequently reported

What is this study about?

This study is about a rare condition on the tongue called lingual microcystic lymphatic malformation (LMLM). This condition causes little cysts (small sacs filled with fluid or blood) to form on the tongue. Even if they are small, these cysts can cause big problems like bleeding, infections, and difficulty with speaking, eating, or breathing. People with LMLM often experience pain and feel self-conscious about their appearance.

The researchers want to see if a cream containing a medicine called sirolimus can help these cysts. Sirolimus is a medicine often used in other ways, but in this study, it will be applied directly to the tongue as a gel. The main goal is to find out if this cream can make the condition less severe after 12 weeks. Doctors will carefully look at photographs of the tongue and score how much the condition has improved.

Patients taking part will also play a crucial role by sharing how their symptoms, like pain, bleeding, and difficulty eating, change over time. They will also talk about their overall well-being and quality of life. This information is very important to understand how the treatment affects daily life. The study aims to gather strong evidence to see if this cream could be a useful new treatment option for LMLM.

Key takeaways

Tests a new cream for a rare tongue condition (LMLM).
Aims to reduce cysts and improve symptoms like pain and bleeding.
Participation involves applying cream, regular photos, and symptom reports.
Study monitors for safety and effectiveness over several months.
May offer symptom relief, but potential side effects are possible.

Who may be eligible?

To join this study, people must be at least 18 years old. The study is open to both men and women.

There may be other specific health requirements or conditions that mean someone cannot join, even if they fit the age and sex criteria. These might include other medical conditions, medicines they are currently taking, or past treatments they have had.

It's important to discuss your full medical history with the study team or your doctor to find out if this study is right for you.

Quick self-check

Am I 18 years old or older?
Am I comfortable applying a cream to my tongue regularly?
Am I able to attend regular appointments for checks and photos?
Do I have lingual microcystic lymphatic malformation (LMLM)?
Am I willing to have blood tests during the study?

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, you'll apply the sirolimus cream to your tongue for 12 weeks. Throughout the study, you'll have regular visits where doctors will take standardised photographs of your tongue each month. You will also fill out questionnaires, rating your symptoms (like pain, bleeding, and ease of eating) from 0 to 10, and share how you feel your condition has changed overall. Your quality of life will also be checked at the start and end of the study.

Doctors will measure the size of your lesion (the affected area) at certain points. They will also do physical exams, check your blood pressure, and take blood samples regularly to ensure the treatment is safe and to check if the medicine is getting into your bloodstream. You'll be monitored for any side effects at each visit. The total duration of active treatment and follow-up will be several months.

Potential risks and benefits

Participating in this study might offer a potential benefit of improving your LMLM symptoms, such as reducing pain, bleeding, and difficulty with daily activities. However, as with any new treatment, there are potential risks. You might experience side effects from the cream, especially on your tongue or in other parts of your body. Doctors will monitor you closely for any local reactions or other health changes through blood tests and physical checks. You are always free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

—
France

Common questions

What is LMLM?

LMLM stands for Lingual Microcystic Lymphatic Malformation, a rare condition that causes small fluid-filled cysts to grow on the tongue, which can cause various problems.

What is sirolimus?

Sirolimus is a medicine that will be used in a cream form in this study. It's hoped it can help reduce the size and symptoms of the cysts on the tongue.

How long will the treatment last?

The main treatment with the cream will last for 12 weeks, but you will be monitored and have check-ups beyond that time.

Will I know if the treatment is working?

Doctors will take photos and you will report on your symptoms regularly. An improvement that reduces your symptoms by even a small amount would be considered a positive step.

Are there any side effects?

Like all medicines, sirolimus cream might have side effects, either where it's applied or elsewhere in your body. The study team will monitor you closely for any issues.