Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
1 other identifier
interventional
52
1 country
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Dec 2009
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2009
CompletedFirst Submitted
Initial submission to the registry
December 10, 2009
CompletedFirst Posted
Study publicly available on registry
December 15, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2011
CompletedFebruary 27, 2012
February 1, 2012
1.2 years
December 10, 2009
February 24, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Rapamycin level
6 months
Complete blood count
6 months
Total cholesterol
6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
6 months
Secondary Outcomes (1)
Reduction in lesion size and appearance
6 months
Study Arms (6)
TSC Placebo Arm
PLACEBO COMPARATORTSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
TSC 1% Arm
EXPERIMENTALTSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
TSC 5% Arm
EXPERIMENTALTSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
NF1 Placebo Arm
PLACEBO COMPARATORNF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
NF1 1% Arm
EXPERIMENTALNF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
NF1 5% Arm
EXPERIMENTALNF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
Interventions
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
The University of Texas Health Science Center
Houston, Texas, 77030, United States
Related Publications (1)
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.
PMID: 22934754DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- 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
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Assistant Professor, Pediatrics-Neurology
Study Record Dates
First Submitted
December 10, 2009
First Posted
December 15, 2009
Study Start
December 1, 2009
Primary Completion
February 1, 2011
Study Completion
June 1, 2011
Last Updated
February 27, 2012
Record last verified: 2012-02