Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Enrollment
- 179
- Locations
- 10
- Primary Endpoint
- Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
Study Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Placebo
Cream only
Intervention: Placebo (Drug)
0.1 % Rapamycin
0.1% Rapamycin cream
Intervention: Rapamycin (Drug)
1% Rapamycin
1% Rapamycin cream
Intervention: Rapamycin (Drug)
Outcomes
Primary Outcomes
Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
Time Frame: baseline, 6 months
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.
Secondary Outcomes
- Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)(baseline, 6 months)
Investigators
Mary Kay Koenig
Associate Professor
The University of Texas Health Science Center, Houston