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Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

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ClinicalTrials.gov Identifier: NCT01526356
Recruitment Status : Completed
First Posted : February 3, 2012
Last Update Posted : June 11, 2015
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Angiofibromas Tuberous Sclerosis Drug: Placebo Drug: Rapamycin Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 177 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Study Start Date : May 2012
Primary Completion Date : August 2014
Study Completion Date : August 2014

Arms and Interventions

Arm Intervention/treatment
Placebo Comparator: Placebo
Cream only
Drug: Placebo
Study cream is applied nightly to the affected areas on the face.
Other Name: 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
Active Comparator: 1% Rapamycin
1% Rapamycin cream
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose

Outcome Measures

Primary Outcome Measures :
  1. Reduction in lesion size and appearance [ Time Frame: 6 months ]
    Photographic, visual, and measurable reduction in the size and severity of the lesions.

Secondary Outcome Measures :
  1. Confirm the lack of systemic uptake of topically applied rapamycin. [ Time Frame: 6 months ]
    Blood levels checked to confirm the lack of systemic rapamycin.

  2. Dermatologic sensitivity at the site of application. [ Time Frame: 6 months ]
    Pain, erythema, or pruritis at the application site.

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01526356

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

Responsible Party: Mary Kay Koenig, Associate Professor, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT01526356     History of Changes
Other Study ID Numbers: HSC-MS-11-0501
Department of Defense USAMRMC ( Other Grant/Funding Number: W81XWH-11-1-0240 )
First Posted: February 3, 2012    Key Record Dates
Last Update Posted: June 11, 2015
Last Verified: June 2015

Keywords provided by Mary Kay Koenig, The University of Texas Health Science Center, Houston:
Tuberous Sclerosis

Additional relevant MeSH terms:
Tuberous Sclerosis
Pathologic Processes
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs