Status:

COMPLETED

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Lead Sponsor:

Dermatology Specialties Limited Partnership

Conditions:

Facial Angiofibroma

Tuberous Sclerosis

Eligibility:

All Genders

6-65 years

Phase:

PHASE2

PHASE3

Brief Summary

The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous...

Detailed Description

Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to ...

Eligibility Criteria

Inclusion

  • Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained
  • Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma
  • An FA severity score of 2 or 3 on the IGA scale
  • Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation
  • Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator

Exclusion

  • Patients who cannot carry out the treatment plan or follow-up assessment
  • Patients with serious skin lesions such as erosions or ulcers
  • Patients with known hypersensitivity to any component of the study product
  • Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
  • Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment
  • Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
  • Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
  • Pregnant or lactating females
  • Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception
  • Patients with immune dysfunction or receiving any form of immunosuppression
  • Patients with severe FA, with a score of 4 on the IGA scale
  • Patients with an FA severity score of less than 2 on the IGA scale

Key Trial Info

Start Date :

July 28 2019

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

September 1 2022

Estimated Enrollment :

107 Patients enrolled

Trial Details

Trial ID

NCT03826628

Start Date

July 28 2019

End Date

September 1 2022

Last Update

September 8 2023

Active Locations (17)

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Page 1 of 5 (17 locations)

1

Phoenix Children's Hospital

Phoenix, Arizona, United States, 85016

2

University of California San Diego

La Jolla, California, United States, 92037

3

All Children's Research Institute

St. Petersburg, Florida, United States, 33701

4

Spectrum Health

Grand Rapids, Michigan, United States, 49503