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Study on Sirolimus for Treating Cervico-facial Lymphatic Malformations in Patients with Poor Prognosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of the medication Rapamycin on a condition known as cervico-facial lymphatic malformations, which are abnormal growths of the lymphatic system in the head and neck area. These malformations can be challenging to treat and may lead to significant health issues. The purpose of the study is to evaluate how well Rapamycin can reduce the size of these malformations.

Participants in the study will receive Rapamycin in the form of an oral solution. The study will monitor the changes in the size of the malformations over time, specifically looking for a reduction in volume. The study will also involve regular check-ups and imaging tests, such as MRI scans, to assess the progress of the treatment. The study aims to determine if Rapamycin can effectively decrease the size of the malformations and improve the symptoms associated with them.

Throughout the study, the safety and any side effects of Rapamycin will be closely monitored. Participants will undergo evaluations at different intervals, including 3 months, 6 months, and 12 months, to track the response to the treatment. The study will also explore how Rapamycin affects certain proteins and factors in the body that are related to the growth of these malformations. This research hopes to provide valuable insights into the potential benefits of Rapamycin for individuals with cervico-facial lymphatic malformations.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will be informed about the trial's objectives and procedures. Consent must be obtained from both parents or guardians.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to evaluate the extent of the cervico-facial lymphatic malformations. This includes a clinical evaluation and imaging studies such as MRI to determine the lesion volume.

  3. Step 3

    Treatment initiation

    The patient will begin treatment with Rapamune 1 mg/mL oral solution, which contains the active substance sirolimus. The medication is administered orally. The dosage and frequency will be determined by the medical team based on the patient's specific needs.

  4. Step 4

    Therapeutic plateau

    The treatment aims to reach a therapeutic plateau, where the optimal response to the medication is achieved. This phase is crucial for evaluating the treatment's effectiveness.

  5. Step 5

    Primary evaluation

    Three months after reaching the therapeutic plateau, the primary evaluation will occur. This involves assessing the reduction in lesion volume using MRI. A reduction of at least 1/5th of the initial volume is considered a positive response.

  6. Step 6

    Secondary evaluations

    Secondary evaluations will be conducted at 6 months and 1 year. These assessments will include MRI to monitor the lesion's evolution and clinical evaluations to document any changes in the lesion's appearance and symptoms.

  7. Step 7

    Adverse reactions monitoring

    Known adverse reactions to the treatment will be monitored monthly. This ensures any side effects are identified and managed promptly.

  8. Step 8

    End of treatment evaluation

    At the end of the treatment period, a final evaluation will be conducted to assess the overall effectiveness of the treatment and any long-term changes in the lesion.

Who can join the trial?

11 criteria

  • Patients must be between 1 and 18 years old.
  • Patients can be of any sex.
  • Patients must have a specific type of growth called a **cervico-facial lymphatic malformation**, which can be on one or both sides of the neck and face, or extend into the chest area.
  • Patients must have ongoing symptoms that cause pain or problems with breathing, swallowing, or speaking, with a **CDS score** below 8. The CDS score is a way to measure the severity of these symptoms.
  • Curative treatment should not be possible or should pose a major risk to function or life. This includes growths that affect large areas of the mouth, throat, or other important structures.
  • Patients over 10 years old must have a **Karnofsky score** above 50%, and those 10 years or younger must have a **Lansky score** above 50%. These scores measure the patient's ability to perform daily activities.
  • Patients must have certain blood test results within specific ranges, including neutrophil count, platelet count, hemoglobin, bilirubin, liver enzymes, albumin, cholesterol, triglycerides, and kidney function.
  • Both parents or guardians must sign an informed consent form.
  • If applicable, a negative pregnancy test is required.
  • Patients must be covered by a Social Security system.
  • If patients have had surgery or a procedure called sclerotherapy, they can be included if they meet the criteria and it has been at least 2 months since the procedure. Some surgeries that do not affect the target area do not require a waiting period.

Who cannot join the trial?

10 criteria

  • Patients who are pregnant or breastfeeding cannot participate.
  • Individuals with a known allergy to the study medication, **Rapamycin**, are excluded. **Rapamycin** is a drug used to treat certain medical conditions.
  • Participants with severe liver or kidney problems are not eligible. The liver and kidneys are important organs that help filter and clean the blood.
  • People who have had another serious illness in the past 6 months are not allowed to join. A serious illness is a condition that can significantly affect health.
  • Anyone currently participating in another clinical trial is excluded. A clinical trial is a research study to test new treatments.
  • Individuals who cannot follow the study procedures or take the study medication as directed are not eligible.
  • Patients with a history of certain types of cancer are excluded. Cancer is a disease where cells in the body grow uncontrollably.
  • People with an active infection that requires treatment are not allowed to participate. An infection is when harmful germs enter the body and cause illness.
  • Individuals with a history of drug or alcohol abuse in the past year are excluded. Abuse means using substances in a harmful way.
  • Anyone with a mental health condition that might interfere with the study is not eligible. Mental health conditions affect a person's mood, thinking, and behavior.
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Investigated drugs

Rapamycin is being studied for its effectiveness in treating cervico-facial lymphatic malformations, which are abnormal formations of lymphatic vessels in the neck and face area. The goal of the trial is to see if Rapamycin can reduce the size of these malformations significantly.

What is already known about the treatment

Rapamycin – Rapamycin is administered orally in the form of tablets or capsules. It is currently being studied for its effectiveness in treating cervico-facial lymphatic malformations with poor prognosis. The main therapeutic indication for Rapamycin in this context is to reduce the volume of these malformations. At the molecular level, Rapamycin works by inhibiting a protein called mTOR, which plays a role in cell growth and proliferation. It is classified pharmacologically as an mTOR inhibitor.

Investigated diseases

Lymphatic Malformations – Lymphatic malformations are non-cancerous growths that occur due to abnormal development of the lymphatic system. These malformations can appear as soft, spongy masses under the skin and are often present at birth or develop in early childhood. They can vary in size and may grow larger over time, especially during periods of rapid growth or infection. In the cervico-facial region, these malformations can cause swelling and may affect nearby structures, potentially leading to functional issues. The growths are typically filled with lymph fluid and can sometimes become infected or bleed. The progression of these malformations can be unpredictable, with some remaining stable while others may increase in size or complexity.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-517769-17-00Protocol code2012_67Estimated enrolment28 patientsSponsorCentre Hospitalier Universitaire De Lille

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