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mTOR inhibitor
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Phase 1
Waitlist Available
Led By Hope Northrup, MD
Research Sponsored by The University of Texas Health Science Center, Houston
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Approved for 5 Other Conditions
All Individual Drugs Already Approved
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.
Eligible Conditions
- Neurofibromatosis
- Cerebral Sclerosis
- Angiofibroma
- Tuberous Sclerosis
- Neurofibroma
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Trial Design
6Treatment groups
Experimental Treatment
Placebo Group
Group I: TSC 5% ArmExperimental Treatment1 Intervention
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
Group II: TSC 1% ArmExperimental Treatment1 Intervention
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
Group III: NF1 5% ArmExperimental Treatment1 Intervention
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
Group IV: NF1 1% ArmExperimental Treatment1 Intervention
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
Group V: TSC Placebo ArmPlacebo Group1 Intervention
TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
Group VI: NF1 Placebo ArmPlacebo Group1 Intervention
NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved
Sirolimus
FDA approved
Find a Location
Logistics
Participation is compensated
You will be compensated for participating in this trial.
Who is running the clinical trial?
Society for Pediatric DermatologyOTHER
8 Previous Clinical Trials
438 Total Patients Enrolled
The University of Texas Health Science Center, HoustonLead Sponsor
957 Previous Clinical Trials
347,800 Total Patients Enrolled
Hope Northrup, MDPrincipal InvestigatorThe University of Texas Health Science Center, Houston
1 Previous Clinical Trials
179 Total Patients Enrolled
Mary Kay Koenig, MDPrincipal InvestigatorThe University of Texas Health Science Center, Houston
3 Previous Clinical Trials
403 Total Patients Enrolled