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

Tracking Information

First Received Date ^ICMJE

February 1, 2012

Last Updated Date

January 28, 2013

Start Date ^ICMJE

May 2012

Estimated Primary Completion Date

August 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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.

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01526356 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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.

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Topical Rapamycin to Erase Angiofibromas in TSC

Official Title ^ICMJE

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

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition ^ICMJE

Angiofibromas
Tuberous Sclerosis

Intervention ^ICMJE

Drug: Placebo
Study cream is applied nightly to the affected areas on the face.

Other Name: Rapamycin
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. Low Dose

Other Name: Rapamycin
Drug: Rapamycin
Study cream is applied nightly to the affected areas on the face. High Dose

Other Name: Rapamycin

Study Arm (s)

Placebo Comparator: Placebo
Cream only

Intervention: Drug: Placebo
Active Comparator: 0.1 % Rapamycin
0.1% Rapamycin cream

Intervention: Drug: Rapamycin
Active Comparator: 1% Rapamycin
1% Rapamycin cream

Intervention: Drug: Rapamycin

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

230

Estimated Completion Date

August 2013

Estimated Primary Completion Date

August 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Both

Ages

Not Provided

Accepts Healthy Volunteers

Contacts ^ICMJE

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

Location Countries ^ICMJE

United States, Australia

Administrative Information

NCT Number ^ICMJE

NCT01526356

Other Study ID Numbers ^ICMJE

HSC-MS-11-0501, Department of Defense USAMRMC

Has Data Monitoring Committee

Yes

Responsible Party

Mary Kay Koenig, The University of Texas Health Science Center, Houston

Study Sponsor ^ICMJE

The University of Texas Health Science Center, Houston

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

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

Information Provided By

The University of Texas Health Science Center, Houston

Verification Date

January 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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