NCT01924273

Novel Treatment for Port Wine Stain Birthmarks

Completed Phase 1 Last updated 28 October 2022

What this trial tests

Phase 1 trial testing Talaporfin sodium in Port-Wine Stain in 7 participants. Completed in 3 July 2017.

Timeline

1 June 2013

Primary endpoint
3 July 2017

3 July 2017

Quick facts

Lead sponsor	University of California, Irvine
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	1 June 2013
Primary completion	3 July 2017
Estimated completion	3 July 2017
Sites	1 location across United States

Drugs / interventions tested

• Talaporfin sodium — full drug profile →

Conditions studied

• Port-Wine Stain — all drugs for Port-Wine Stain →

Sponsor

University of California, Irvine

Who can join

18 and older, any sex, with Port-Wine Stain. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Port wine stain blanching
  Time frame: up to 12 weeks
  The outcome measure will be PWS blanching as assessed by Diffuse Reflectance Imaging/Laser Speckle Imaging/Spatial Frequency Domain Imaging.

Sponsor's own description

Port wine stains Birthmark are congenital, progressive vascular malformations of the skin that occur in approximately 0.7% of newborns. Approximately 1.5 million individuals in the United States and 32 million people worldwide have Port wine stains birthmarks. Two-thirds of these malformations occur on the face. Personality development of virtually all patients is adversely affected as a result of the negative reaction of others to a "marked" person. Detailed studies have documented lower self-esteem and difficulties with interpersonal interactions in Port wine stains patients. Port wine stains are initially flat and red, but with time, they tend to darken to purple and become thickened as vascular nodules develop. This thickening occurs in approximately two-thirds of lesions and further disfigures the facial features of many patients.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Leveraging a Sturge-Weber Gene Discovery: An Agenda for Future Research.
   Comi AM, Sahin M, Hammill A, Kaplan EH, et al · · 2016 · cited 13× · PMID 27268758 · DOI 10.1016/j.pediatrneurol.2015.11.009