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Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex | Clinical Trials | Clareo Health

COMPLETEDPHASE2/PHASE3

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

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

NCT03826628•Dermatology Specialties Limited Partnership

View on ClinicalTrials.gov

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).

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 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).

Eligibility

Age

6 - 65 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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

Exclusion Criteria

•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
+2 more criteria

Locations (17)

Phoenix Children's Hospital

Phoenix, Arizona, United States

University of California San Diego

La Jolla, California, United States

All Children's Research Institute

St. Petersburg, Florida, United States

Spectrum Health

Grand Rapids, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

University of Virginia

Charlottesville, Virginia, United States

Children's Health Queensland

Brisbane, Queensland, Australia

Fakultni nemocnice Brno

Brno, Czechia

Bethesda Children's Hospital of the Hungarian Reformed Church

Budapest, Hungary

University of Pécs

Pécs, Hungary

Key Dates

Start Date

July 28, 2019

Primary Completion Date

September 1, 2022

Completion Date

September 1, 2022

Last Updated

September 8, 2023

Enrollment

107

ACTUAL participants

Conditions

Facial AngiofibromaTuberous Sclerosis

Interventions

rapamycin

DRUG

placebo

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 8, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Study of Skin Tumors in Tuberous Sclerosis

Familial Investigations of Childhood Cancer Predisposition

Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling

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