Topical Rapamycin to Erase Angiofibromas in TSC

• Status: Completed
• Trial end date: 2014-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.

The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The University of Texas Health Science Center, Houston
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Subjects must be willing and able to comply with all trial requirements.

• Subject has a diagnosis of TSC and has visible facial angiofibromas.

• Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods .

Exclusion Criteria:

• Subject is currently receiving therapy with Rapamycin.

• 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 a clinical trial involving an investigational drug.

• Subject has a known hypersensitivity to either the vehicle or Rapamycin.

• Subject is a pregnant or nursing female.

• Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.

• Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.