A Randomized Trial to Study Combined Pulsed Dye Laser and Rapamycin Treatment of Port Wine Stain Birthmarks.

The researchers want to collect data on safety and efficacy of combined pulsed dye laser and rapamycin to improve fading/blanching of port wine stain birthmarks as compared to pulsed dye laser alone, which is the current standard of care.

This single center pilot and feasibility study will have a target enrollment of 40 port wine stain subjects at the Beckman Laser Institute and Medical Clinic, University of California, Irvine.

Study Overview

• Status: Completed
• Conditions: Port Wine Stain
• Intervention / Treatment: Drug: Laser and rapamycin versus laser alone

Detailed Description

The study will offer two different approaches to the treatment of port wine stain birthmarks.

Study Group Number 1: Port wine stain treated by the pulsed dye laser alone, which is the current standard of care: 20 subjects.

Study Group Number 2: Port wine stain treated by combined pulsed dye laser and rapamycin: 20 subjects.

The researchers want to collect data on safety and efficacy of combined pulsed dye laser and rapamycin to improve fading/blanching of port wine stain birthmarks as compared to pulsed dye laser alone, which is the current standard of care. Twenty subjects will have their entire port wine stain birthmark treated by pulsed dye laser alone. Twenty subjects will have their entire port wine stain birthmark treated by combined pulsed dye laser and rapamycin, which is a drug that prevents the formation of blood vessels in the skin. Safety will be evaluated by searching for any local effects such as injury to the skin or systemic effects such as abnormal blood and/or urine studies. Efficacy will be determined by objectively measuring and comparing the port wine stain fading/blanching responses for the two study groups.

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92612
      • Beckman Laser Institute and Medical Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Port wine stain suitable for comparison testing.
• Age > 13 years of age or older; minor will be accompanied in the room by parents or guardians during laser treatment.
• Apparent good health as documented by medical history.
• Ability to understand and carry out subject instructions.
• Women of childbearing potential must have a negative urinary pregnancy test prior to being started on rapamycin.
• Women of child bearing potential must agree to use a medically acceptable method of contraception throughout the study and for 3 months following discontinuation of rapamycin.

Exclusion Criteria:

• Inability to understand and carry out instructions.
• Pregnancy.
• Abnormal blood or urine tests
• History of cancer.
• History of high cholesterol, lipids or liver disease.
• Allergy to macrolide drugs (e.g., erythromycin).
• Any therapy within the previous two months to the proposed port wine stain treatment sites.
• Current participation in any other investigational drug evaluation.
• Concurrent use of known photosensitizing drugs.
• Concurrent use of immunosuppressive drugs or steroids.
• Concurrent use of any of the following medications: antifungals, antiepileptics, protease inhibitors, cimetidine, cisapride, clarithromycin, dannzol, diltiazem, erythromycin, metoclopramide, rifabutin, rifampin, rifapetine, troleandomycin, or verapamil.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Laser and rapamycin versus laser alone	Drug: Laser and rapamycin versus laser alone; Laser and rapamycin versus laser alone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Improved port wine stain fading/blanching after laser treatment	Efficacy will be determined by objectively measuring and comparing the port wine stain fading/blanching responses for subjects treated with combined pulsed dye laser and rapamycin versus pulsed dye laser alone	24 weeks

Collaborators and Investigators

• Sponsor: University of California, Irvine
• Collaborators: Pfizer; Beckman Laser Institute University of California Irvine
• Investigators: Principal Investigator: J. Stuart Nelson, M.D.,Ph.D., Beckman Laser Institute and Medical Clinic, University of California Irvine

Study record dates

Study Major Dates

• Study Start: December 1, 2008
• Primary Completion (ACTUAL): January 1, 2016
• Study Completion (ACTUAL): January 1, 2016

Study Registration Dates

• First Submitted: January 27, 2009
• First Submitted That Met QC Criteria: January 27, 2009
• First Posted (ESTIMATE): January 28, 2009

Study Record Updates

• Last Update Posted (ACTUAL): November 1, 2022
• Last Update Submitted That Met QC Criteria: October 28, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: port wine stain
• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Skin Abnormalities; Hemangioma; Neoplasms, Vascular Tissue; Hemangioma, Capillary; Port-Wine Stain; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: 20086383

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED