NCT07436611

Rapamycin Dose-Ranging Efficacy Study in Port Wine Stains

Rapamycin Dose-Ranging Efficacy Study: A Phase II, Proof of Concept, Double-blind, Placebo-controlled, Randomized, Parallel-group, Dose-response Comparison of the Effects of Different Strengths of Rapamycin Cream Applied Topically in Subjects Diagnosed With Port Wine Stains

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: AFT Pharmaceuticals, Ltd. · Industry
• Sex: All
• Age: 10 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to learn if the use of rapamycin cream can be used as a treatment together with pulsed dyed laser in treating port wine stain birthmarks. The main question it aims to answer is: Will rapamycin cream and laser treatment show a greater improvement in appearance of port wine birthmarks, compared to treatment with placebo cream and laser? Researchers will compare two concentrations of rapamycin cream (0.6% or 1.0%) with placebo treatment to see if appearance is improved following 12 weeks of treatment. Participants will receive laser treatment of their port wine birthmark and then apply the rapamycin or placebo cream daily for 12 weeks. Patients will visit the clinic every 4 weeks for checkups and tests.

Detailed description

This is a single-centre, double-blind, randomized withdrawal design, parallel group, placebo-controlled, dose-response clinical study investigating the efficacy and safety of two concentrations of topical rapamycin cream in the treatment of port wine stain as an adjunct to pulsed dyed laser (PDL) in paediatric patients. 30 participants will be enrolled into the study. The study hypothesis is that topical rapamycin, as an adjunct to PDL, will show a dose-dependent increase in the blanching of port wine birthmarks, compared to PDL with placebo after 12 weeks. Eligible participants will undergo PDL treatment of their entire port wine birthmark. Patients that respond positively to PDL within 5 days will be enrolled and randomized, before the first application of the study drug. Participants will be randomly assigned to one of three possible treatments in a 1:1:1 ratio: * Rapamycin cream, topical (0.6% rapamycin) * Rapamycin cream, topical (1.0% rapamycin) * Placebo The stain will be divided into two parts. One part will be treated only with PDL and the other will be treated with both PDL and the assigned cream. Cream will be applied once daily for 12 weeks. During this phase of the study, four clinical visits will occur, one every 4 weeks for 12 weeks. Assessment of the extent and severity of birthmark size and colour will be performed at randomization, at each clinical visit, and at follow-up using photography and percentage improvement ratings. Safety will be assessed by recording all adverse events experienced and testing blood rapamycin concentration at prior to treatment and at the last clinical visit. Follow-up will be performed 28 days after the last cream application. An extra PDL session will be optionally provided at the follow-up appointment. If PDL treatment occurs at the follow-up visit, a further follow-up phone call will occur 7±2 days after the second PDL session to assess concomitant medications and adverse events.

Conditions

• Port Wine Stains

Interventions

Type	Name	Description
DRUG	Rapamycin cream	Application of rapamycin cream to port wine birthmark
PROCEDURE	Pulsed dyed laser	Pulsed dyed laser (PDL) treatment of port wine birthmark

Timeline

• Start date: 2026-04-01
• Primary completion: 2027-09-01
• Completion: 2027-10-01
• First posted: 2026-02-27
• Last updated: 2026-02-27

Locations

2 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study