Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

February 24, 2012 updated by: Mary Kay Koenig, The University of Texas Health Science Center, Houston

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months.

The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

52

Phase

  • Phase 1

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

  • United States
    • Texas
      • Houston, Texas, United States, 77030
        • The University of Texas Health Science Center

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

13 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Subject is willing and able to comply with all trial requirements
  • Subject is male or female and over 13 years of age
  • Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
  • Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial

Exclusion Criteria:

  • Subject is currently receiving therapy with rapamycin or sirolimus
  • 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 any clinical trial involving an investigational drug
  • Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
  • Subject is a pregnant or nursing female

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
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
PLACEBO_COMPARATOR: TSC Placebo Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
Drug: Skincerity
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
EXPERIMENTAL: TSC 1% Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
Drug: Skincerity plus sirolimus/rapamycin
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
EXPERIMENTAL: TSC 5% Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
Drug: Skinercity plus sirolimus/rapamycin
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
PLACEBO_COMPARATOR: NF1 Placebo Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
Drug: Skincerity
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
EXPERIMENTAL: NF1 1% Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
Drug: Skincerity plus sirolimus/rapamycin
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
EXPERIMENTAL: NF1 5% Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
Drug: Skinercity plus sirolimus/rapamycin
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Total cholesterol
Time Frame: 6 months
6 months
Rapamycin level
Time Frame: 6 months
6 months
Complete blood count
Time Frame: 6 months
6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Reduction in lesion size and appearance
Time Frame: 6 months
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

Collaborators

Society for Pediatric Dermatology

Investigators

  • Principal Investigator: Hope Northrup, MD, The University of Texas Health Science Center, 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

December 1, 2009

Primary Completion (ACTUAL)

February 1, 2011

Study Completion (ACTUAL)

June 1, 2011

Study Registration Dates

First Submitted

December 10, 2009

First Submitted That Met QC Criteria

December 14, 2009

First Posted (ESTIMATE)

December 15, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

February 27, 2012

Last Update Submitted That Met QC Criteria

February 24, 2012

Last Verified

February 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HSC-MS-09-0259

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.

Clinical Trials on Tuberous Sclerosis

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe