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Trial record 7 of 13 for:    Open Studies | "Tuberous Sclerosis"
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Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

This study is currently recruiting participants.
Verified January 2013 by The University of Texas Health Science Center, Houston
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: January 28, 2013
Last verified: January 2013
History of Changes
  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:

U.S. FDA Resources

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 2013
Estimated Primary Completion Date: August 2013 (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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01526356

Contacts
Contact: Patti L Tate, RCP, CCRP 713-500-5659 ext XXXX patti.l.tate@uth.tmc.edu
Contact: Mary Kay Koenig, MD 713-500-7142 ext XXXX mary.k.koenig@uth.tmc.edu

Locations
United States, Alabama
University of Alabama at Birmingham Recruiting
Birminham, Alabama, United States, 35294
Contact: Cheryl Hall, LPN     256-533-0833     chall@uab.edu    
Contact: Martina Bebin, MD     256-533-0833     ebebin@uab.edu    
Principal Investigator: Martina Bebin, MD            
Sub-Investigator: Bruce Korf, MD, PhD            
United States, Massachusetts
Herscot Center for Adults and Children with TSC Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Susana Camposano, MD     617-726-0241     scamposano@partners.org    
Contact: Elizebeth Theile, MD     617-726-0241     etheile@partners.org    
Principal Investigator: Elizabeth Theile, MD            
Sub-Investigator: Susana Camposano, MD            
United States, Minnesota
Clinic Without Walls Recruiting
Saint Paul, Minnesota, United States, 55102-2697
Contact: Geraldine Pira, BSN     651-241-5058     gpira@msepilepsy.net    
Contact: Michael Frost, MD         mfrost@mnepilepsy.net    
Principal Investigator: Michael Frost, MD            
United States, Texas
The University of Texas Medical School at Houston Recruiting
Houston, Texas, United States, 77030
Contact: Patti L Tate, RCP, CCRP     713-500-5659 ext XXXX     patti.l.tate@uth.tmc.edu    
Contact: Mary Kay Koenig, MD     713-500-7142 ext XXXX     mary.k.koenig@uth.tmc.edu    
Principal Investigator: Mary Kay Koenig, MD            
Sub-Investigator: Hope Northrup, MD            
Sub-Investigator: Joshua Samuels, MD, MPH            
Sub-Investigator: Adelaide Hebert, MD            
Sub-Investigator: John Slopis, MD            
Australia, New South Wales
Sydney Children's Hospital Recruiting
Sydney, New South Wales, Australia
Contact: Vanessa Bonett     61-29382-0512     vanessa.bonett@sesiahs.health.nsw.gov.au    
Contact: David Mowat, MD         david.mowat@sesiah.health.nsw.au    
Principal Investigator: David Mowat, MD            
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: January 28, 2013
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:
Tuberous Sclerosis
Sclerosis
Angiofibroma
Pathologic Processes
Hamartoma
Neoplasms
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
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents

ClinicalTrials.gov processed this record on May 23, 2013