Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

September 30, 2020 updated by: Mary Kay Koenig, The University of Texas Health Science Center, Houston

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

179

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia
        • Sydney Children's Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama at Birmingham
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Mattel Children's Hospital
      • Oakland, California, United States, 94609
        • Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Herscot Center for Adults and Children with TSC Massachusetts General Hospital
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102-2697
        • Clinic Without Walls
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Texas
      • Dallas, Texas, United States, 75219
        • Texas Scottish Rite Hospital
      • Houston, Texas, United States, 77030
        • The University of Texas Medical School at Houston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Cream only
Drug: Placebo
Study cream is applied nightly to the affected areas on the face.
Other Names:
  • Rapamycin
Active Comparator: 0.1 % Rapamycin
0.1% Rapamycin cream
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose
Active Comparator: 1% Rapamycin
1% Rapamycin cream
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
baseline, 6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
Time Frame: baseline, 6 months
baseline, 6 months
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
Time Frame: baseline, 6 months
The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
baseline, 6 months
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
Time Frame: baseline, 6 months
The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
baseline, 6 months
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
Time Frame: baseline, 6 months
The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
baseline, 6 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Events of Dermatologic Sensitivity at the Site of Application
Time Frame: 6 months
Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.
6 months
Number of Participants With Systemic Uptake of Topically Applied Rapamycin
Time Frame: 6 months
Blood levels checked to confirm the lack of systemic rapamycin.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The University of Texas Health Science Center, Houston

Investigators

  • Principal Investigator: Mary Kay Koenig, MD, The University of Texas Medical School at Houston
  • Principal Investigator: Hope Northrup, MD, The University of Texas Medical School at Houston

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2012

Primary Completion (Actual)

August 1, 2014

Study Completion (Actual)

August 1, 2014

Study Registration Dates

First Submitted

February 1, 2012

First Submitted That Met QC Criteria

February 2, 2012

First Posted (Estimate)

February 3, 2012

Study Record Updates

Last Update Posted (Actual)

October 26, 2020

Last Update Submitted That Met QC Criteria

September 30, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HSC-MS-11-0501
  • Department of Defense USAMRMC (Other Grant/Funding Number: W81XWH-11-1-0240)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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