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

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Brief Title

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

Official Title

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

Brief Summary

      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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Rapamycin level

Secondary Outcome

 Reduction in lesion size and appearance

Condition

Tuberous Sclerosis

Intervention

Skincerity

Study Arms / Comparison Groups

 TSC Placebo Arm
Description:  TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

52

Start Date

December 2009

Completion Date

June 2011

Primary Completion Date

February 2011

Eligibility Criteria

        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
      

Gender

All

Ages

13 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Mary Kay Koenig, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01031901

Organization ID

HSC-MS-09-0259


Responsible Party

Principal Investigator

Study Sponsor

The University of Texas Health Science Center, Houston

Collaborators

 Society for Pediatric Dermatology

Study Sponsor

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


Verification Date

February 2012