Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma
About this trial
This is an interventional treatment trial for Tuberous Sclerosis focused on measuring Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus
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
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
Placebo Comparator
Experimental
Experimental
Placebo Comparator
Experimental
Experimental
TSC Placebo Arm
TSC 1% Arm
TSC 5% Arm
NF1 Placebo Arm
NF1 1% Arm
NF1 5% Arm
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