Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01526356
First received: February 1, 2012
Last updated: April 3, 2014
Last verified: April 2014
  Purpose

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 Intervention Phase
Angiofibromas
Tuberous Sclerosis
Drug: Placebo
Drug: Rapamycin
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by The University of Texas Health Science Center, Houston:

Primary Outcome Measures:
  • Reduction in lesion size and appearance [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Photographic, visual, and measurable reduction in the size and severity of the lesions.


Secondary Outcome Measures:
  • Confirm the lack of systemic uptake of topically applied rapamycin. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    Blood levels checked to confirm the lack of systemic rapamycin.

  • Dermatologic sensitivity at the site of application. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    Pain, erythema, or pruritis at the application site.


Estimated Enrollment: 230
Study Start Date: May 2012
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Other Name: Rapamycin
Active Comparator: 1% Rapamycin
1% Rapamycin cream
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose
Other Name: Rapamycin

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01526356

Locations
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
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
  More Information

No publications provided

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
Study First Received: February 1, 2012
Last Updated: April 3, 2014
Health Authority: United States: Federal Government

Keywords provided by The University of Texas Health Science Center, Houston:
Angiofibroma
Tuberous Sclerosis
Rapamycin
Sirolimus
mTOR

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

ClinicalTrials.gov processed this record on November 20, 2014