Port Wine Stain
Conditions
Interventions
Trade Name: Rapamune
Product Name: sirolimus
Pharmaceutical Form: Cutaneous solution
INN or Proposed INN: SIROLIMUS
CAS Number: 53123-88-9
Current Sponsor code: N/A
Other descriptive name: Rapamune
Co
Sponsors
None listed
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Subject has provided informed consent; • Subject is = 18 years of age at time of screening; • Subject has an extra-facial homogenous PWS; • The PWS is large enough in size to fit one of the templates (at least 11 cm x 3 cm or 7 cm x 5 cm); • Subject has not received any laser treatment of the PWS in the last 3 months (in the treatment area); • The PWS has a minimal erythema grading score of 3 (on a 4 point scale) in the opinion of the investigator; • Screening blood safety values are within normal parameters or regarded as not clinically significant in the opinion of the investigator. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: • PWS with a nodular/hypertrophic component in the treatment area; • PWS on cosmetically unacceptable locations in the opinion of the investigator; • For women: pregnant or breast feeding during the treatment period; • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using adequate contraceptive measures; Effective contraception is defined as either: o Barrier method of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicide. The following methods are considered more effective than the barrier method and are also acceptable: o Total abstinence (when this is in line with the preferred and usual lifestyle of the subject). o Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before participating in the study. o Male sterilization (at least 6 months prior to screening). For female subjects on the study, the vasectomized male partner should be the sole partner for that subject. o Use of oral, injected or implanted hormonal methods of contraception or other forms, intrauterine device (IUD) or intrauterine system (IUS) NOTE: Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago. • Subject is known to have immune deficiency, or is immune compromised (including immunosuppression induced by medication); • Known allergy to sirolimus or other constituents of the study medication; • Incapacitated subjects; • Any medical or psychiatric condition which, in the investigator’s opinion, would preclude the participant from adhering to the protocol or completing the study per protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate and compare the efficacy of: 1) Pulsed Dye Laser (PDL) treatment followed by topical sirolimus application after Erbium Yag (Er:Yag) laser ablation of the stratum corneum 2) PDL treatment followed by topical sirolimus application without Er:Yag laser ablation of the stratum corneum 3) PDL treatment only 4) Sirolimus application only ;Secondary Objective: - To evaluate the improvement in quality of life; - To evaluate patient discomfort and pain experienced during and following treatment; - To evaluate patient-reported symptoms and side-effects; - To assess systemic sirolimus exposure after local application by measuring serum concentrations; - To evaluate treatment satisfaction. ;Primary end point(s): Percentage clearance of the treated area assessed colorimetrically (Minolta colorimeter).;Timepoint(s) of evaluation of this end point: 12 weeks, 46 weeks, 94 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Percentage clearance of the treated area assessed by standardized digital colour image analysis. - Percentage clearance of the treated area assessed by photographic evaluation by an expert panel. - To evaluate the improvement in quality of life (DLQI); - Patient discomfort and pain experienced during and following treatment (VAS); - Patient-reported symptoms and side-effects; - Systemic sirolimus exposure; - Treatment satisfaction (0-100 scale). ;Timepoint(s) of evaluation of this end point: 12 weeks, 46 weeks, 94 weeks | — |
Countries
Netherlands
Outcome results
None listed