Summary

for people ages 6-65 (full criteria)
at La Jolla, California and other locations
study started
estimated completion
Doris Trauner

Description

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 Sclerosis Complex (TSC).

Official Title

A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over

Details

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 be well tolerated.

The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).

Keywords

Facial Angiofibroma Tuberous Sclerosis Angiofibroma Sclerosis Facies Sirolimus Everolimus rapamycin

Eligibility

You can join if…

Open to people ages 6-65

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

You CAN'T join if...

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

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92037 United States
  • Phoenix Children's Hospital not yet accepting patients
    Phoenix Arizona 85016 United States

Lead Scientist

  • Doris Trauner
    Recall Hcomp, Neurosciences. Authored (or co-authored) 51 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Dermatology Specialties Limited Partnership
ID
NCT03826628
Phase
Phase 2/3
Study Type
Interventional
Last Updated