Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

Completed · Phase 1 · Has a placebo group

Conditions studied: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma

In brief

This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months. The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.

Key facts

Study ID

NCT01031901

Run by

The University of Texas Health Science Center, Houston

People needed

52

Starts

2009-12-01

Expected to finish

2011-06-01

Last updated by the study team

2012-02-27

Who can join

Age: 13 and older. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Subject is willing and able to comply with all trial requirements
• Subject is male or female and over 13 years of age
• Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
• Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial

You may not qualify if…

• Subject is currently receiving therapy with rapamycin or sirolimus
• Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction
• Subject is currently participating in or has participated within the last 30 days in any clinical trial involving an investigational drug
• Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
• Subject is a pregnant or nursing female

Where it is running

• The University of Texas Health Science Center — Houston, Texas, United States

Full record on ClinicalTrials.gov

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