Port Wine StainsPhase 2NCT07436611
Rapamycin Dose-Ranging Efficacy Study in Port Wine Stains
A Phase 2 study testing Rapamycin cream and Pulsed dyed laser in people with Port Wine Stains, run by AFT Pharmaceuticals, Ltd. at 2 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Port Wine Stains
- What is being tested?
- Rapamycin cream and Pulsed dyed laser
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 13 Nov 2026to Oct 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female patients aged ≤ 10 years on the day informed consent is obtained
- Patients presenting either facial, neck, or upper trunk port-wine stains
- Port wine stain size of at least 5 cm tall x 2.5 cm wide (2x1 inches)
- Patients (or their legal representatives) capable of understanding the explanation of the clinical trial, and who give written informed consent for participation
- Patients (or their legal representatives) able to maintain patient diaries following the instructions of the investigator or sub-investigator
- Patients (or their guardians) are willing and able to follow instructions to only apply cream to part of their port-wine stain
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- Skin type classified as either I, II, III or IV on the Fitzpatrick scale
- Patients who are indicated for PDL treatment and accept this treatment
- Patients who respond to PDL with purpura or blanching
Who cannot join
- Skin type classified as either V or VI on the Fitzpatrick scale
- Patients with PWS in extremities and/or acral areas
- Patients unwilling or unable to carry out the treatment plan or follow-up assessment
- Patients with serious skin lesions such as erosions or ulcers
- Patients with known hypersensitivity to any component of the study product
- Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
Show all 13Show fewer
- Patients who have received laser therapy or surgical therapy to treat PWS within 3 months prior to trial enrolment
- Patients who have received 5 or more PDL treatments previously
- Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
- Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
- Pregnant or lactating females
- Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception*
- Patients with immune dysfunction or receiving any form of immunosuppression
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
United States1 site
Driscoll Children's Hospital
Corpus Christi, Texas, 78411
Erin Richmond, MS, RN, CCRP, 361-694-5978, erin.richmond@dchstx.org
Denise Metry, MD
Spain1 site
Sant Joan de Déu University Hospital
Barcelona, Catalonia, 08950
Study Coordinator - Unitat d'Investigacio Clinica, +34 670989833, mateu.torres@sjd.es
Marta Ivars, MD
About this study
Official title: 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
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?
Read more
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.
Read the 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
- Being tested
- Rapamycin cream, Pulsed dyed laser
- Keywords
- sirolimus, rapamycin, adjunct therapy, proof of concept, phase 2, dose ranging
- Medical subject headings
- Hemangioma, Capillary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rapamycin creamDrug
Application of rapamycin cream to port wine birthmark
Pulsed dyed laserProcedure or surgery
Pulsed dyed laser (PDL) treatment of port wine birthmark
| Study group | Role | What they receive |
|---|---|---|
| 0.6% rapamycin | Experimental | 0.6% rapamycin cream applied daily Receives: Drug: Rapamycin cream; Procedure: Pulsed dyed laser |
| 1.0% rapamycin cream | Experimental | 1.0% rapamycin cream applied daily Receives: Drug: Rapamycin cream; Procedure: Pulsed dyed laser |
| Placebo cream | Placebo comparator | Placebo cream applied daily Receives: Procedure: Pulsed dyed laser |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinic Objective improvement rating at week 12
The average percentage improvement (0-100% with higher scores being a better outcome) of port wine stain appearance in the cream treated area from baseline to week 12, or at last visit if early withdrawal/discontinuation occurs, as assessed by the principal investigator during the clinic visit.
Measured: From before laser treatment to the end of treatment after 12 weeks of cream application
Secondary outcomes (3)
Subjective improvement rating (all timepoints)
Subjective (patient or parent/caregiver) improvement rating (0-100% with higher scores being a better outcome) of port wine stain from baseline after 0, 4, 8 and 12 weeks of treatment.
Measured: From before laser treatment to the end of treatment after 12 weeks of cream application
Clinic Objective improvement rating (all timepoints)
Clinic Objective (investigator) improvement rating (0-100% with higher scores being a better outcome) in port wine stain from baseline after 0, 4, 8 and 12 weeks of treatment
Measured: From before laser treatment to the end of treatment after 12 weeks of cream application
Photographic Objective improvement rating (all timepoints)
Photographic Objective improvement rating (0-100% with higher scores being a better outcome), assessed by a committee of three dermatologists, in port wine stain from baseline after 0, 4, 8 and 12 weeks of treatment
Measured: From before laser treatment to the end of treatment after 12 weeks of cream application
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start13 Nov 2026 (estimated)
- Primary completionOct 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Port Wine StainsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- A Phase II, proof of concept, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison, with a secondary self-controlled assessment
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AFT Pharmaceuticals, Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ioana Stanescu, Study director, AFT Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07436611
- Sponsor's protocol ID
- AFT-RAP-01
- Other IDs
- 2025-520821-19-00, 2025
- Status last verified
- Jun 2026
- First submitted
- 22 Feb 2026
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07436611
- Acronym / sponsor ID
- AFT-RAP-01
- Conditions
- Port Wine Stains
- Keywords
- sirolimus, rapamycin, adjunct therapy, proof of concept, phase 2, dose ranging
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- AFT Pharmaceuticals, Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Nov 2026 (estimated)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 27 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- A Phase II, proof of concept, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison, with a secondary self-controlled assessment
| Arm | Type | Receives |
|---|---|---|
| 0.6% rapamycin | Experimental | Drug: Rapamycin cream, Procedure: Pulsed dyed laser0.6% rapamycin cream applied daily |
| 1.0% rapamycin cream | Experimental | Drug: Rapamycin cream, Procedure: Pulsed dyed laser1.0% rapamycin cream applied daily |
| Placebo cream | Placebo comparator | Procedure: Pulsed dyed laserPlacebo cream applied daily |
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Male and female patients aged ≤ 10 years on the day informed consent is obtained
- Patients presenting either facial, neck, or upper trunk port-wine stains
- Port wine stain size of at least 5 cm tall x 2.5 cm wide (2x1 inches)
- Patients (or their legal representatives) capable of understanding the explanation of the clinical trial, and who give written informed consent for participation
- Patients (or their legal representatives) able to maintain patient diaries following the instructions of the investigator or sub-investigator
Key exclusion criteria (13 listed)
- Skin type classified as either V or VI on the Fitzpatrick scale
- Patients with PWS in extremities and/or acral areas
- Patients unwilling or unable to carry out the treatment plan or follow-up assessment
- Patients with serious skin lesions such as erosions or ulcers
- Patients with known hypersensitivity to any component of the study product
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rapamycin creamApplication of rapamycin cream to port wine birthmark | Drug | 0.6% rapamycin, 1.0% rapamycin cream |
| Pulsed dyed laserPulsed dyed laser (PDL) treatment of port wine birthmark | Procedure or surgery | 0.6% rapamycin, 1.0% rapamycin cream, Placebo cream |
Primary outcome
Clinic Objective improvement rating at week 12
Time frame: From before laser treatment to the end of treatment after 12 weeks of cream application
The average percentage improvement (0-100% with higher scores being a better outcome) of port wine stain appearance in the cream treated area from baseline to week 12, or at last visit if early withdrawal/discontinuation occurs, as assessed by the principal investigator during the clinic visit.
3 secondary outcomes
Subjective improvement rating (all timepoints)
Time frame: From before laser treatment to the end of treatment after 12 weeks of cream application
Clinic Objective improvement rating (all timepoints)
Time frame: From before laser treatment to the end of treatment after 12 weeks of cream application
Photographic Objective improvement rating (all timepoints)
Time frame: From before laser treatment to the end of treatment after 12 weeks of cream application
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ioana Stanescu study director
AFT Pharmaceuticals
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rapamycin cream and Pulsed dyed laser in people with Port Wine Stains, run by AFT Pharmaceuticals, Ltd. at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Laura Boddington, PhD (+6494880232 ext. 735, laura.boddington@aftpharm.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinic Objective improvement rating at week 12 (time frame: From before laser treatment to the end of treatment after 12 weeks of cream application). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.