Clinical Trial: Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II, Randomized, Triple Blind, Intra-individually Placebo-controlled Clinical Trial to Assess the Efficacy and Safety of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge

Brief 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.

Detailed Summary: Patients with SWS will be treated with 2 sessions of PDL in the lateral part of the CM separated by an interval of 6 weeks and with 1% topical rapamycin or placebo in the superior or inferior half, both applied once a day for 12 weeks. The clinical response will be analyzed using a morphologic and chromatographic computerised system and with spectrometry. Histological response will be evaluated also. For that purpose, we will make 4 biopsies, one in each quadrant (quadrant treated with PDL and placebo, quadrant treated with PDL and rapamycin, quadrant treated only with rapamycin and quadrant treated only with placebo)
Sponsor: Clinica Universidad de Navarra, Universidad de Navarra

Current Primary Outcome:

  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 6 [ Time Frame: Baseline, Week 6 ]
    Change Outcome Measure
  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 12 [ Time Frame: Baseline, Week 12 ]
    Change Outcome Measure
  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 18 [ Time Frame: Baseline, Week 18 ]
    Change Outcome Measure
  • Histological response at 12 weeks. [ Time Frame: 12 weeks ]
    Efficacy Outcome Measure


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Adverse events at baseline [ Time Frame: At the beginning of the intervention ]
  • Adverse events at 6 weeks [ Time Frame: 6 weeks after the beginning of the intervention ]
  • Adverse events at 12 weeks [ Time Frame: 12 weeks after the beginning of the intervention ]
  • Adverse events at 18 weeks [ Time Frame: 18 weeks after the beginning of the intervention ]
  • Total blood cholesterol level (mg/dL) at baseline. [ Time Frame: At the beginning of the intervention ]
  • Total blood cholesterol level (mg/dL) at 6 weeks. [ Time Frame: 6 weeks after the beginning of the intervention ]
  • Blood concentration of triglycerides (mg/dL) at baseline. [ Time Frame: At the beginning of the intervention ]
  • Blood concentration of triglycerides (mg/dL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention ]
  • Blood concentration of hemoglobin (g/dL) at baseline. [ Time Frame: At the beginning of the intervention ]
  • Blood concentration of hemoglobin (g/dL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention ]
  • Blood count of leukocytes (number of cells/mL) at baseline. [ Time Frame: At the beginning of the intervention. ]
  • Blood count of leukocytes (number of cells/mL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ]
  • Blood platelet count (number of platelets/mL) at baseline. [ Time Frame: At the beginning of the intervention. ]
  • Blood concentration of rapamycin (ng/ml) at baseline. [ Time Frame: At the beginning of the intervention. ]
  • Blood concentration of rapamycin (ng/ml) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ]
  • Blood platelet count (number of platelets/mL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ]


Original Secondary Outcome: Same as current

Information By: Clinica Universidad de Navarra, Universidad de Navarra

Dates:
Date Received: April 18, 2013
Date Started: January 2011
Date Completion:
Last Updated: March 4, 2014
Last Verified: October 2013