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

NCT02080624 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2014-03-06

No results posted yet for this study

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.

Conditions

  • Sturge- Weber Syndrome

Interventions

DRUG

Drug: Topical Rapamycin

After signing this consent form, you will be asked to undergo some screening tests or procedures to find out if you can be in the research study. A medical history, which involves questions about your health history, any medications you are taking or plan to take, any allergies and the treatments you received for your CM. A physical exam, during which you will be asked about any problems that you might be having. Additionally, your clinician will check your vital signs (blood pressure, heart rate, weight and height). The doctor will also evaluate your performance status, which indicates how much your illness affects your activity level. Blood tests, which will be done to make sure your hemogram, triglyceride and cholesterol levels are normal. A pregnancy test for females will be done to check that you are not pregnant. If theses tests show that you are eligible to participate in the research study, you will begin the study treatment.

Sponsors & Collaborators

  • Clinica Universidad de Navarra, Universidad de Navarra

    lead OTHER

Principal Investigators

  • Maider Pretel, MD PhD · Clinica Universidad de Navarra

  • Leyre Aguado, MD PhD · Clinica Universidad de Navarra

  • Laura Marqués, MD · Clinica Universidad de Navarra

Study Design

Allocation
NA
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-01-31
Primary Completion
2012-09-30
Completion
2012-12-31

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02080624 on ClinicalTrials.gov