Topical Rapamycin to Erase Angiofibromas in TSC

The University of Texas Health Science Center, Houston179 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

179

Conditions

Angiofibromas Tuberous Sclerosis

Interventions

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (10)

University of Alabama at Birmingham

Birmingham, Alabama, United States

UCLA Mattel Children's Hospital

Los Angeles, California, United States

Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center

Oakland, California, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Herscot Center for Adults and Children with TSC Massachusetts General Hospital

Boston, Massachusetts, United States

Clinic Without Walls

Saint Paul, Minnesota, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Texas Scottish Rite Hospital

Dallas, Texas, United States

The University of Texas Medical School at Houston

Houston, Texas, United States

Sydney Children's Hospital

Sydney, New South Wales, Australia

Outcomes

Primary Outcomes

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score

Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.