Health TrialsbyHealthPanel

Rapamycin Dose-Ranging Efficacy Study in Port Wine Stains

ConditionPort Wine Stains
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 10
SponsorAFT Pharmaceuticals, Ltd.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Rapamycin cream

    Drug

    Application of rapamycin cream to port wine birthmark

  • Pulsed dyed laser

    Procedure/Surgery

    Pulsed dyed laser (PDL) treatment of port wine birthmark

Treatment groups

30 Participants
are divided into 3 treatment groups
Group A: 0.6% rapamycinExperimental treatment 2 interventions
Group B: 1.0% rapamycin creamExperimental treatment 2 interventions
Group C: Placebo creamPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
From before laser treatment to the end of treatment after 12 weeks of cream application

Secondary outcomes

1

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.

Time frame
From before laser treatment to the end of treatment after 12 weeks of cream application
2

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

Time frame
From before laser treatment to the end of treatment after 12 weeks of cream application
3

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

Time frame
From before laser treatment to the end of treatment after 12 weeks of cream application

Other outcomes

1

Subjective improvement rating change at follow-up

Subjective (patient or parent/caregiver) improvement rating (0-100% with higher scores being a better outcome) change of cream treated area from baseline at follow-up visit

Time frame
From before laser treatment to follow-up visit (16 weeks)
2

Subjective improvement rating (post-treatment)

Subjective (patient or parent/caregiver) improvement rating (0-100% with higher scores being a better outcome) change of cream treated area between week 12 and follow-up

Time frame
4 weeks between end of treatment and follow-up visit
3

Clinic Objective improvement rating change at follow-up

Clinic Objective (investigator) improvement rating (0-100% with higher scores being a better outcome) change of cream treated area from baseline at follow-up visit

Time frame
From before laser treatment to follow-up visit (16 weeks)
4

Clinic Objective improvement rating (post-treatment)

Clinic Objective (investigator) improvement rating (0-100% with higher scores being a better outcome) change of cream treated area between week 12 and follow-up

Time frame
4 weeks between end of treatment and follow-up visit

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AFT Pharmaceuticals, Ltd.

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: