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

Primary Purpose

Tuberous Sclerosis, Neurofibromatoses, Angiofibroma

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Skincerity
Skincerity plus sirolimus/rapamycin
Skinercity plus sirolimus/rapamycin
Sponsored by
The University of Texas Health Science Center, Houston
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tuberous Sclerosis focused on measuring Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

  • The University of Texas Health Science Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Placebo Comparator

Experimental

Experimental

Placebo Comparator

Experimental

Experimental

Arm Label

TSC Placebo Arm

TSC 1% Arm

TSC 5% Arm

NF1 Placebo Arm

NF1 1% Arm

NF1 5% Arm

Arm Description

TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas

TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas

TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas

NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas

NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas

NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas

Outcomes

Primary Outcome Measures

Rapamycin level
Complete blood count
Total cholesterol
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)

Secondary Outcome Measures

Reduction in lesion size and appearance

Full Information

First Posted
December 10, 2009
Last Updated
February 24, 2012
Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Society for Pediatric Dermatology
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1. Study Identification

Unique Protocol Identification Number
NCT01031901
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
Study Type
Interventional

2. Study Status

Record Verification Date
February 2012
Overall Recruitment Status
Completed
Study Start Date
December 2009 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
June 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Society for Pediatric Dermatology

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Keywords
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
52 (Actual)

8. Arms, Groups, and Interventions

Arm Title
TSC Placebo Arm
Arm Type
Placebo Comparator
Arm Description
TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
Arm Title
TSC 1% Arm
Arm Type
Experimental
Arm Description
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
Arm Title
TSC 5% Arm
Arm Type
Experimental
Arm Description
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
Arm Title
NF1 Placebo Arm
Arm Type
Placebo Comparator
Arm Description
NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
Arm Title
NF1 1% Arm
Arm Type
Experimental
Arm Description
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
Arm Title
NF1 5% Arm
Arm Type
Experimental
Arm Description
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
Intervention Type
Drug
Intervention Name(s)
Skincerity
Intervention Description
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
Intervention Type
Drug
Intervention Name(s)
Skincerity plus sirolimus/rapamycin
Intervention Description
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
Intervention Type
Drug
Intervention Name(s)
Skinercity plus sirolimus/rapamycin
Intervention Description
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
Primary Outcome Measure Information:
Title
Rapamycin level
Time Frame
6 months
Title
Complete blood count
Time Frame
6 months
Title
Total cholesterol
Time Frame
6 months
Title
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Reduction in lesion size and appearance
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mary Kay Koenig, MD
Organizational Affiliation
The University of Texas Health Science Center, Houston
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Hope Northrup, MD
Organizational Affiliation
The University of Texas Health Science Center, Houston
Official's Role
Principal Investigator
Facility Information:
Facility Name
The University of Texas Health Science Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
22934754
Citation
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.
Results Reference
derived

Learn more about this trial

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

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