Clinical Trial: Treatment of Resistant Port-wine Stains With Bosentan and Pulsed Dye Laser: a Pilot Study

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Treatment of Port-wine Stains by Bosentan in Addition to Pulsed Dye Laser (PDL) in Children or Young Adults Who Previously Failed to Respond to PDL Alone: a Monocentric Pi

Brief Summary: Pulsed dye laser (PDL) is the gold standard treatment for port-wine stains (PWS). However, the outcomes are highly variable due to the new angiogenesis occurring after laser irradiation. Studies suggest that endothelin is involved in the neoangiogenesis that occurred after treatment of port-wine stains by PDL. The main objective of this pilot clinical trial is to evaluate the effectiveness and safety of an inhibitor of endothelin orally taken, the Bosentan, following PDL treatment. Four patients with facial port-wine stain resistant to the PDL treatment will be included. The treatment by Bosentan (2 mg/kg twice daily, maximum 62,5 mg twice daily) will be given one day before the PDL irradiation and continued for 14 days. Only one test area of PWS will be treated with PDL. The primary outcome measure will be an important or complete improvement (Investigator Global Assessment 3 or 4) between treated area and non treated one, 14 days after the end of the treatment which corresponds to one month after the laser PDL session. The evaluation will been performed on standardized pictures by 2 independent physicians blinded to the region treated or not.

Detailed Summary: Pulsed dye laser (PDL) is the gold standard treatment for port-wine stains (PWS). However, the outcomes are highly variable due to the new angiogenesis occurring after laser irradiation. Studies suggest that endothelin is involved in the neoangiogenesis that occurred after treatment of port-wine stains by PDL . The main objective of this pilot clinical trial is to evaluate the effectiveness and safety of an inhibitor of endothelin orally taken, the Bosentan, following PDL treatment. Four patients with facial port-wine stain resistant to the PDL treatment will be included. The treatment by Bosentan (2 mg/kg twice daily, maximum 62,5 mg twice daily) will be given one day before the PDL irradiation (maximum surface 100 cm²) and continued for 14 days. Only one test area of PWS will be treated with PDL. The primary outcome measure will be an important or complete improvement (Investigator Global Assessment 3 or 4) between treated area and non treated one, 14 days after the end of the treatment which corresponds to one month after the laser PDL session. The evaluation will been performed on standardized pictures by 2 independent physicians blinded to the region treated or not.
Sponsor: Centre Hospitalier Universitaire de Nice

Current Primary Outcome: Investigator Global Assessment [ Time Frame: at 1 month after the start of treatment with bosentan and Pulsed Dye Laser ]

The primary outcome measure will be an important or complete improvement (Investigator Global Assessment) between treated area and non treated one, at 1 month after the start of treatment by bosentan and Pulsed Dye Laser


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Patient satisfaction [ Time Frame: 1 time at 1 month after the start of treatment with bosentan and Pulsed Dye Laser ]
    The secondary outcome measure will be the satisfaction of the patients (or their parents), or the patient alone if major, about the efficiency and the safety of the treatments at 1 month after the start of treatment by bosentan and Pulsed Dye Laser. A visual analogical scale will be used.
  • Side effects [ Time Frame: 3 times : First time the day of start of bosentan. Second time, one day after the start of treatment with bosentan. The third time, 30 days after the start of treatment with bosentan and pulsed dye laser ]
    Frequency, severity and occurrence of side effects will be noted.
  • Satisfaction of the patients of the treatment using visual analogical scale [ Time Frame: 1 time at 1 month after the start of treatment by bosentan and Pulsed Dye Laser ]
    The secondary outcome measure will be the satisfaction of the patients (or their parents), or the patient alone if major, about the safety of the treatments at 1 month after the start of treatment with bosentan and Pulsed Dye Laser


Original Secondary Outcome: Same as current

Information By: Centre Hospitalier Universitaire de Nice

Dates:
Date Received: March 21, 2014
Date Started: April 2014
Date Completion: February 2015
Last Updated: December 15, 2014
Last Verified: December 2014