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mTOR inhibitor

NF1 1% Arm for Tuberous Sclerosis

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 6 months
Awards & highlights

Study 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
  • Tuberous Sclerosis
  • Neurofibromatosis
  • Angiofibroma
  • Neurofibroma

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Complete blood count
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
Rapamycin level
+1 more
Secondary outcome measures
Reduction in lesion size and appearance

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
526 Total Patients Enrolled
The University of Texas Health Science Center, HoustonLead Sponsor
910 Previous Clinical Trials
323,520 Total Patients Enrolled
3 Trials studying Tuberous Sclerosis
669 Patients Enrolled for Tuberous Sclerosis
Hope Northrup, MDPrincipal InvestigatorThe University of Texas Health Science Center, Houston
1 Previous Clinical Trials
179 Total Patients Enrolled
1 Trials studying Tuberous Sclerosis
179 Patients Enrolled for Tuberous Sclerosis

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
Drugs in R&DJournal
Topical Rapamycin Therapy to Alleviate the Cutaneous Manifestations of Tuberous Sclerosis Complex
Koenig, Mary Kay, Adelaide A. Hebert, Joan Roberson, Joshua Samuels, John Slopis, Audrey Woerner, and Hope Northrup. 2012. “Topical Rapamycin Therapy to Alleviate the Cutaneous Manifestations of Tuberous Sclerosis Complex”. Drugs in R&d. Springer Science and Business Media LLC. doi:10.2165/11634580-000000000-00000.
clinicaltrials.govStudy
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Mary Kay Koenig 2009. "Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01031901.
~3 spots leftby May 2025
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