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Long‑Term Effects of Sirolimus on Volume and Color of Port‑Wine Stain in Patients with Vascular Malformation Undergoing Surgery and Laser Therapy

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What is this study about?

A Vascular malformation known as a port-wine stain can affect the lip, causing it to become enlarged (a condition called macrocheilia) and giving the skin a reddish or purplish color. The study will give participants an oral medication called sirolimus, which is taken either as a liquid solution or as coated tablets, and will also involve standard surgery and laser therapy, a treatment that uses focused light to improve the appearance of the skin.

The purpose of this phase II study is to find out whether taking sirolimus after surgery can lead to a lasting reduction in the size of the affected lip and a change in the color of the birthmark, while also checking safety and how the condition impacts quality of life. Participants will start the medication after their surgical procedure and will be followed for several years, with periodic visits to measure the lip’s size with a simple ruler, to assess color changes using a four‑step scale, and to complete questionnaires that ask about daily activities and comfort.

1 initial enrollment and baseline assessment

after you are accepted into the study, you will complete a baseline assessment that includes measurement of the volume of the port‑wine macrocheilia (pwm) using a caliper in three dimensions, and evaluation of the color intensity of the port‑wine stain.

your quality of life will also be recorded using the ovama face and neck score.

2 start sirolimus treatment

you will begin oral treatment with sirolimus branded as rapamune. the medication may be provided as a 1 mg/mL oral solution or as 2 mg coated tablets.

the prescribed dose is 5 mg each day, taken once daily. the frequency is daily and the duration is intended for up to 60 months (5 years) unless a physician decides otherwise.

3 surgical intervention

after a short period on sirolimus, you will undergo the planned surgery related to the vascular malformation.

the surgery is performed while you continue the daily sirolimus dose.

4 laser therapy sessions

following surgery, you will receive laser therapy sessions as scheduled by the study team.

laser treatments are carried out while you remain on sirolimus.

5 regular follow‑up visits

you will attend follow‑up visits at regular intervals (for example, every 3 months) during the 60‑month study period.

at each visit the volume of pwm and the color of the stain will be re‑measured, and any side effects of sirolimus will be recorded.

quality of life will be reassessed using the same ovama score at designated time points.

6 final 60‑month assessment

at the end of the 60‑month period, a comprehensive assessment will be performed to determine the change in pwm volume, color intensity, and quality of life compared with the baseline.

the data will be used to evaluate the long‑term effect of sirolimus after surgery and laser therapy.

Who Can Join the Study?

  • Be a male or female patient who has a port‑wine stain (PWS) on the middle (V2) or lower (V3) part of the face, and also has a thickened upper or lower lip (called PWM).
  • Have a port‑wine stain that has either gotten larger or stayed the same during the two months before joining the study.
  • Be older than 5 years but younger than 25 years.
  • Have a positive result on a genetic test for the GNAQ R183 change, which looks for a specific DNA alteration linked to the condition.
  • Have an overall health rating of ASA I (healthy) or ASA II (mild systemic disease) according to the American Society of Anesthesiologists classification.
  • Show adequate bone‑marrow function, meaning:
    • Hemoglobin higher than 10.0 g/dL (a measure of red blood cells).
    • Neutrophils higher than 1,500 per mm³ (a type of white blood cell that fights infection).
    • Platelets higher than 100,000 per mm³ (cells that help blood clot).
  • Have laboratory results within safe limits:
    • Total bilirubin no more than 1.5 times the normal upper limit (bilirubin is a substance processed by the liver).
    • ALT and AST (liver enzymes) no more than three times the normal upper limit.
    • Creatinine no more than 1.5 times the normal upper limit, or if higher, a 24‑hour creatinine clearance of at least 60 mL/min (a test of kidney function).
  • For women who could become pregnant, have a negative urine pregnancy test.
  • Provide written and verbal informed consent, meaning you agree to take part after understanding the study.

Who Cannot Join the Study?

  • If you are unlikely to follow the study rules and take the medication as instructed.
  • If you have stomach or intestinal problems that could stop the medicine from being absorbed, such as ulcer disease, frequent nausea, vomiting, moderate or worse diarrhea, conditions where the gut does not absorb nutrients properly, or if part of your small intestine has been surgically removed.
  • If you are allergic (have a hypersensitivity) to drugs that are similar to the study medicine, such as everolimus or temsirolimus.
  • If you are allergic to soy or peanuts, because the oral solution contains soy.
  • If you have another serious or uncontrolled health problem that could react badly with the study drug.
  • If you do not understand or read Norwegian, or if your legal representative does not.
  • If you have previously been treated with medicines that block the PI3K or mTOR pathways (these are types of drugs used for other conditions).
  • If you are taking other medicines that cannot be used together with sirolimus.
  • If you have had a cancer diagnosis within the past five years, except for a non‑invasive cervical lesion called carcinoma in situ.
  • If you have a weakened immune system (are immunocompromised), including having HIV infection.
  • If you have a large amount of protein in your urine (more than 3 grams in a 24‑hour collection), a condition called proteinuria.
  • If you are pregnant, could become pregnant, or are a female partner of a male participant and are not using reliable birth control, and you would need to continue birth control for at least 12 weeks after stopping the study drug.
  • If you are currently breastfeeding.
  • If you have serious heart problems, such as unstable chest pain (angina), dangerous heart rhythm problems (ventricular arrhythmia), valve disease that affects heart function, a heart attack within the last six months, heart muscle disease (cardiomyopathy), or a family history of a heart rhythm condition called long or short QT.

Where you can join this trial?

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Other Sites

Site Name City Country Status
Oslo University Hospital HF Oslo Norway

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Norway Norway
Not yet recruiting
01.10.2026

Trial locations

Investigated Drugs:

Rapamune oral solution is a liquid medicine that contains the active ingredient sirolimus. Sirolimus works by gently calming the immune system, which can help reduce abnormal tissue growth. In this study the liquid form is taken by mouth, allowing the drug to be absorbed through the digestive tract. The purpose of giving this medication is to see if it can help shrink the enlarged, reddish area on the lip (port‑wine macrocheilia) after the patients have had surgery.

Rapamune coated tablets are a pill form of the same medication, also containing sirolimus. Like the liquid version, the tablet is swallowed and the drug is absorbed into the body to modulate the immune response. Using a tablet provides an alternative way for participants to take the medicine while the study evaluates its long‑term effect on the size and color of the port‑wine lesion.

Surgery in this trial refers to a planned operation on the lip to remove or reduce the excess tissue caused by the port‑wine macrocheilia. The surgical procedure creates a more normal shape and size, and the study then observes whether sirolimus helps keep the improvement stable over many years.

Laser therapy is a non‑invasive treatment that uses focused light beams to target the reddish skin discoloration of the port‑wine stain. The laser helps break down the pigment and can improve the appearance of the skin. In the study, laser treatment is combined with sirolimus medication to assess whether the drug enhances the lasting benefits of the laser on color and volume.

Investigated Diseases:

Vascular malformation – A vascular malformation is an abnormal cluster of blood vessels that is present at birth and grows proportionally with the child. Over time, the lesion may become larger or change shape as the surrounding tissues develop. The condition typically progresses slowly, with the affected area potentially becoming more noticeable as the individual ages.

Trial ID:
2024-511937-37-01
Protocol code:
PLASS-Q
Trial Phase:
Therapeutic exploratory (Phase II)

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