Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Facial Angiofibroma Clinical Trial

Official title: Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Status Completed

Clinical Trial Summary

The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment.

Clinical Trial Description

This is an open label, prospective single center study. Patients that are seen in our regional Tuberous Sclerosis Complex Clinic and have facial angiofibromas that would benefit from this cream and who wish to be involved in this study would be enrolled. We anticipate enrolling 12-15 patients the first year. Patients will have pictures of their facial angiofibromas taken immediately prior to entry into the study. They will then be prescribed 0.1% rapamune topical formulation to apply twice daily to their skin for the first two weeks and then once daily. If level is above 0, will re-check serum lipids, cbc, and repeat levels every three-four months and monitor the patient for symptoms. Patients will return at one month and three months to obtain serum rapamune levels along with pictures, then they will be seen intermittently throughout the first year with photographs of their face taken at the one month visit, 3 month visit, six month visit and twelve month visit. We will ask the parents about any side effects from the medication during the entire time patients are using the topical rapamune. We will monitor the facial lesions as well. We will assess for skin sensitivity, pain, erythema, or pruritus. If skin irritation results from the drug, then we will stop use of the drug. Parents will sign an informed consent allowing their child's picture to be taken and allowing us to summarize the data for publication at the end of this study.

This study will be the first study using a consistent formulation to make a 0.1% rapamune cream, and this will be performed under the direction of myself and Dr. Almoazen in the University of Tennessee Health Science Center(UTHSC) College of Pharmacy compounding laboratory. Only patients who are thought to be reliable for the follow up visits and to use the cream appropriately will be enrolled in the study. This study meets the FDA criteria for an Investigational New Drug(IND) exemption.

The adverse events noted in the package insert for rapamune cream are all based on systemic administration of the medication, primarily to adults with other diseases. In our clinical experience, with topical use there is no absorption and we have not noted any side effects. ;

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Angiofibroma
• Facial Angiofibroma
• Facies
• Sclerosis
• Tuberous Sclerosis

• NCT number: NCT01853423
• Study type: Interventional
• Source: Le Bonheur Children's Hospital
• Status: Completed
• Phase: Phase 1
• Start date: October 2013
• Completion date: July 2016