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mTOR inhibitor

NF1 1% Arm for Neurofibromatosis

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
  • Tuberous Sclerosis Complex
  • Angiofibroma
  • Tuberous Sclerosis
  • Neurofibroma

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

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
946 Previous Clinical Trials
344,533 Total Patients Enrolled
Hope Northrup, MDPrincipal InvestigatorThe University of Texas Health Science Center, Houston
1 Previous Clinical Trials
179 Total Patients Enrolled
~3 spots leftby Nov 2025
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