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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01031901
Recruitment Status : Completed
First Posted : December 15, 2009
Last Update Posted : February 27, 2012
Sponsor:
Collaborator:
Society for Pediatric Dermatology
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston

Tracking Information
First Submitted Date  ICMJE December 10, 2009
First Posted Date  ICMJE December 15, 2009
Last Update Posted Date February 27, 2012
Study Start Date  ICMJE December 2009
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 14, 2009)
  • Rapamycin level [ Time Frame: 6 months ]
  • Complete blood count [ Time Frame: 6 months ]
  • Total cholesterol [ Time Frame: 6 months ]
  • Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis) [ Time Frame: 6 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 14, 2009)
Reduction in lesion size and appearance [ Time Frame: 6 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Official Title  ICMJE 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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Tuberous Sclerosis
  • Neurofibromatoses
  • Angiofibroma
  • Neurofibroma
Intervention  ICMJE
  • Drug: Skincerity
    Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or 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
  • 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
Study Arms  ICMJE
  • Placebo Comparator: TSC Placebo Arm
    TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
    Intervention: Drug: Skincerity
  • Experimental: TSC 1% Arm
    TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
    Intervention: Drug: Skincerity plus sirolimus/rapamycin
  • Experimental: TSC 5% Arm
    TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
    Intervention: Drug: Skinercity plus sirolimus/rapamycin
  • Placebo Comparator: NF1 Placebo Arm
    NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
    Intervention: Drug: Skincerity
  • Experimental: NF1 1% Arm
    NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
    Intervention: Drug: Skincerity plus sirolimus/rapamycin
  • Experimental: NF1 5% Arm
    NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
    Intervention: Drug: Skinercity plus sirolimus/rapamycin
Publications * Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.

*   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 Completed
Actual Enrollment  ICMJE
 (submitted: February 24, 2012)
52
Original Estimated Enrollment  ICMJE
 (submitted: December 14, 2009)
60
Actual Study Completion Date  ICMJE June 2011
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01031901
Other Study ID Numbers  ICMJE HSC-MS-09-0259
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
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 Society for Pediatric Dermatology
Investigators  ICMJE
Principal Investigator: Mary Kay Koenig, MD The University of Texas Health Science Center, Houston
Principal Investigator: Hope Northrup, MD The University of Texas Health Science Center, Houston
PRS Account The University of Texas Health Science Center, Houston
Verification Date February 2012

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