Clinical Trial: Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

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

Official Title: Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T

Brief Summary: This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period. The evaluation will be done by 3D photography measuring volume with the LIFEVIZ Micro system.

Detailed Summary: Qualifying subjects will have a diagnosis of Neurofibromatosis 1 and have disfiguring cutaneous lesions that can be measured by photography. The subjects will have photographs of the target lesions,biopsies of the lesions,and safety blood laboratory tests at 3 time points (baseline, 3 months and 6 months which is end of treatment). Everolimus will be taken orally for 6 months. Subjects will visit the clinic monthly for an exam and adverse event evaluation. Laboratory testing will be done at these visits if determined necessary by the PI.
Sponsor: The University of Texas Health Science Center, Houston

Current Primary Outcome: 3D Photographic Measurement of surface volume [ Time Frame: 6 months ]

Photographs of selected lesions to measure surface volume.


Original Primary Outcome: Same as current

Current Secondary Outcome: Determination if orally administered Afinitor is safe in patients [ Time Frame: 6 months ]

Lack of Grade 3-4 adverse events during the trial period.


Original Secondary Outcome: Same as current

Information By: The University of Texas Health Science Center, Houston

Dates:
Date Received: December 12, 2014
Date Started: February 2015
Date Completion:
Last Updated: May 10, 2016
Last Verified: May 2016