NCT03826628

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 107 (actual)
• Sponsor: Dermatology Specialties Limited Partnership · Industry
• Sex: All
• Age: 6 Years – 65 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Facial Angiofibroma
• Tuberous Sclerosis

Interventions

Type	Name	Description
DRUG	rapamycin	Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
DRUG	placebo	Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

Timeline

• Start date: 2019-07-28
• Primary completion: 2022-09-01
• Completion: 2022-09-01
• First posted: 2019-02-01
• Last updated: 2023-09-08
• Results posted: 2023-08-14

Locations

17 sites across 9 countries: United States, Australia, Czechia, Hungary, New Zealand, Serbia, Slovakia, Spain, Taiwan

Regulatory

• FDA-regulated drug study