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Study on the Effects of Sirolimus Cream for Patients with Port Wine Stains

Verified siteRegistered drugNo placebo

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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 a treatment for port wine stains, which are birthmarks caused by swollen blood vessels that create a reddish-purple discoloration on the skin. The treatment being tested is a cream containing sirolimus, also known by the code name AFT-RAP-01. The cream is applied directly to the skin, and the study will compare two different strengths of the cream, 0.5% and 1.0%, to see how well they work in improving the appearance of port wine stains.

The purpose of the study is to determine if the sirolimus cream can improve the look of port wine stains when used alongside a type of laser therapy called PDL therapy. Participants in the study will be randomly assigned to receive either the sirolimus cream or a placebo cream, and the effects will be observed over a period of 12 weeks. The study aims to see if the cream is effective and well-tolerated by those using it.

Throughout the study, changes in the color and intensity of the port wine stains will be monitored using photographs and a special color measurement system. Participants will have their progress checked at the start, and then again at weeks 4, 8, and 12. The study will also gather feedback from participants on how they feel about the changes in their port wine stains over time.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit and baseline assessment
During the initial visit, a baseline assessment is conducted. This includes taking standardized clinical photographs to measure the extent and intensity of the port wine stain (PWS) color.
The patient receives instructions on how to apply the rapamycin cream and maintain a patient diary.
Step 2
Treatment phase
The patient applies the rapamycin cream topically to the designated area of the PWS. The cream is available in two strengths: 0.5% and 1.0%.
The application is done following the instructions provided, and the treatment continues for a period of 12 weeks.
Step 3
Follow-up assessments
Follow-up assessments occur at weeks 0, 4, 8, and 12. During these visits, the change in the extent and intensity of the PWS color is measured using a colorimetry system through standardized clinical photographs.
Both subjective and objective improvement ratings of the PWS are recorded at each follow-up visit.
Step 4
Final assessment
At the end of the 12-week treatment period, a final assessment is conducted. This includes a last set of standardized clinical photographs to evaluate the primary efficacy endpoint, which is the change in PWS color from baseline.
If early withdrawal or discontinuation occurs, the final assessment is conducted at the last visit.

Who can join the trial?

7 criteria

Patients must be **male or female** and aged **3 months to 18 years** on the day they agree to participate.
Patients should have **port-wine stains** on their **face, neck, trunk, or upper arm**. Port-wine stains are a type of birthmark that looks like a red or purple mark on the skin.
The **port-wine stain** must be at least **36 square centimeters** in size, which is about the size of a square that is 6 centimeters on each side.
Patients or their legal representatives must be able to understand the details of the clinical trial and provide **written consent** to participate. This means they agree to join the study after understanding what it involves.
Patients or their legal representatives must be able to keep a **patient diary** as instructed by the study team. A patient diary is a record of certain activities or symptoms that the patient keeps during the study.
Patients must be willing and able to follow instructions to apply the cream only to a part of their **port-wine stain**.
The patient's **skin type** should be classified as **I, II, III, or IV** on the **Fitzpatrick scale**. The Fitzpatrick scale is a way to describe how different skin types react to sunlight.

Who cannot join the trial?

4 criteria

Individuals who do not have a diagnosis of port wine stains cannot participate. Port wine stains are a type of birthmark that looks like a red or purple mark on the skin.
Participants must be within the specified age range, which includes children and adults.
Both males and females are eligible to participate.
Individuals who are considered part of a vulnerable population may not be eligible. A vulnerable population includes groups of people who might need special protection or care.

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Verified sites

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Verified
Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain

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Investigated drugs

Rapamycin Cream: This is a topical cream used in the study to improve the appearance of port wine stains on the skin. The cream is applied directly to the affected area. The study is testing two different strengths of the cream to see how effective they are in enhancing the results of a specific laser therapy known as PDL therapy. The goal is to determine if the cream can help reduce the visibility of the skin condition and to ensure that it is safe and well-tolerated by the participants.

Sirolimus

What is already known about the treatment

Rapamycin Cream – This medication is applied topically in the form of a cream. It is currently being studied in clinical trials to assess its effectiveness in improving the appearance of port wine stains, a type of skin discoloration. The main therapeutic indication is for individuals with port wine stains, particularly following pulsed dye laser (PDL) therapy. Rapamycin works by inhibiting a specific protein involved in cell growth, which may help reduce the appearance of these skin marks. It is classified as an immunosuppressant and is being evaluated for its potential benefits in dermatological applications.

Investigated diseases

Port Wine Stains – Port wine stains are a type of birthmark caused by an abnormal development of blood vessels in the skin. They appear as flat, pink, red, or purple marks and can vary in size. These marks are present at birth and tend to grow proportionally with the child. Over time, they may become darker and thicker. Port wine stains are usually found on the face, neck, arms, or legs. They are generally harmless but can sometimes be associated with other medical conditions.