Clinical trial NCT02317679: Treatment of Resistant Port-wine Stains With Bosentan and Pulsed Dye Laser: a Pilot Study
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 Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Port-wine Stains
Lead sponsor: Centre Hospitalier Universitaire de Nice
Sponsor class: OTHER
First posted: 2014-12-16
Enrollment: 4
Sex: ALL
Minimum age (years): 7
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 description: 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.

Eligibility criteria: Inclusion Criteria:

* Children of 7 years old and over or adults aged under 60
* with a resistant port-wine stain after treatment by PDL will be included in this pilot study.
* The agreement of the parents and the child or the patient alone if major will be required.
* Subjects have to be registered to the social security.
* An informed consent will have to be signed by the parents or the patient if of a suitable age or the patient alone if major.
* An efficient contraception will be mandatory if the patient is female and an age to give birth.

Exclusion criteria:

* Hypersensitivity to the Bosentan or to one of its excipients.
* Mild to severe liver disease corresponding to the Child-Pugh Score B or C.
* Serum levels of ASAT and/or ALAT greater three times the upper limit of normal. - Concurrent use of cyclosporine.
* Pregnancy.

Primary outcomes:
measure: Investigator Global Assessment
timeFrame: at 1 month after the start of treatment with bosentan and Pulsed Dye Laser
Secondary outcomes:
measure: Patient satisfaction
timeFrame: 1 time at 1 month after the start of treatment with bosentan and Pulsed Dye Laser
measure: Side effects
timeFrame: 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
measure: Satisfaction of the patients of the treatment using visual analogical scale
timeFrame: 1 time at 1 month after the start of treatment by bosentan and Pulsed Dye Laser
Other outcomes:
Locations:
city: Nice
country: France
facility: CHU de Nice - Dermatologie - Hôpital Archet
state: Alpes-maritimes
Study officials:
affiliation: CHU De Nice, Dermatologie, Hôpital de l'archet 151 route de st-antoine de ginestière 06200 nice
name: PASSERON Thierry, Phd
role: PRINCIPAL_INVESTIGATOR
References: