Port-wine Stains Clinical Trial
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
| Verified date | January 2018 |
| Source | Centre Hospitalier Universitaire de Nice |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 4 |
| Est. completion date | February 2015 |
| Est. primary completion date | September 2014 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 7 Years to 60 Years |
| Eligibility |
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. |
| Country | Name | City | State |
|---|---|---|---|
| France | CHU de Nice - Dermatologie - Hôpital Archet | Nice | Alpes-maritimes |
| Lead Sponsor | Collaborator |
|---|---|
| Centre Hospitalier Universitaire de Nice |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Investigator Global Assessment | 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 | at 1 month after the start of treatment with bosentan and Pulsed Dye Laser | |
| Secondary | Patient satisfaction | 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. | 1 time at 1 month after the start of treatment with bosentan and Pulsed Dye Laser | |
| Secondary | Side effects | Frequency, severity and occurrence of side effects will be noted. | 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 | |
| Secondary | Satisfaction of the patients of the treatment using visual analogical scale | 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 | 1 time at 1 month after the start of treatment by bosentan and Pulsed Dye Laser |