Naevus flammeus Trials in Disease

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

This clinical trial is investigating the effectiveness of a topical cream containing sirolimus (also referred to as rapamycin or AFT-RAP-01) for treating port wine stains. The study is designed to evaluate whether applying this cream directly to the skin can improve the appearance of these birthmarks, particularly when used alongside pulsed dye laser (PDL) therapy.

Who can participate:

• Children and young people between 3 months and 18 years of age
• Those with port wine stains located on the face, neck, trunk, or upper arm
• The affected area must be at least 36 square centimeters in size (approximately 6 centimeters on each side)
• Participants must have skin types classified as I, II, III, or IV on the Fitzpatrick scale, which describes how skin reacts to sunlight
• Patients or their legal representatives must be able to provide written consent after understanding the study details
• Participants must be willing to keep a patient diary and follow instructions for applying the cream to only a portion of their port wine stain

Who cannot participate:

• Individuals who do not have a confirmed diagnosis of port wine stains
• Those outside the specified age range
• People who are considered part of a vulnerable population that may require special protection or care

What the study involves:

The trial will test two different strengths of sirolimus cream: 0.5% and 1.0%. Participants will be randomly assigned to receive either the active cream or a placebo cream. The treatment period lasts 12 weeks, during which participants apply the cream to their skin as instructed.

The study follows a structured timeline:

1. At the initial visit, standardized photographs are taken to measure the color and extent of the port wine stain. Participants receive detailed instructions on cream application and how to maintain their patient diary.
2. During the 12-week treatment phase, participants apply the cream to the designated area of their port wine stain according to the provided instructions.
3. Follow-up assessments occur at weeks 0, 4, 8, and 12. At each visit, photographs are taken and color measurements are performed using a special colorimetry system to track changes in the appearance of the birthmark.
4. At the end of the 12 weeks, a final assessment evaluates the overall change in the port wine stain's appearance compared to the beginning of the study.

Throughout the study, both objective measurements (using photographs and color analysis) and subjective ratings (based on how participants and researchers perceive improvement) are recorded. Participants also provide feedback on how they feel about the changes in their condition over time.

Investigational drug:

The medication being tested is Rapamycin Cream, a topical treatment applied directly to the skin. Rapamycin, also known as sirolimus, works by inhibiting a specific protein involved in cell growth. It is classified as an immunosuppressant and is being evaluated for its potential to reduce the visibility of port wine stains when used in combination with laser therapy. The study aims to determine not only whether the cream is effective but also whether it is safe and well-tolerated by children and young people.