Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome
Status:
Completed
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
Sturge-Weber syndrome (SWS) is a rare congenital neuro-cutaneous disorder considered as a
rare or orphan disease. SWS is characterized by a capillary vascular malformation (CM)
localized on the skin of the face, eyes and central nervous system. Given the localization
and the extent of the CM, children with SWS are particularly prone to developing severe
psychological problems. The standard treatment for CM is pulsed dye laser (PDL) although in
these cases whitening of the lesion is rarely achieved. Combining topical rapamycin, a
specific inhibitor of the mammalian target of rapamycin, with PDL is hypothesised to be a
good therapeutic option in these patients.
Phase:
Phase 2
Details
Lead Sponsor:
Clinica Universidad de Navarra, Universidad de Navarra