NCT03363763

Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

Phase 2 Multi Center Prospective Rand. Double Blind Placebo Cont. Parallel Design Study to Evaluate Safety & Efficacy of Topical Sirolimus for Cutaneous Angiofibromas in Subjects W/ Tuberous Sclerosis Complex Followed by Opt. Open Label

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Aucta Pharmaceuticals, Inc · Industry
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to evaluate the safety and efficacy of sirolimus (0.2% and 0.4% formulations) and its vehicle when applied topically once daily for 12 weeks for the treatment of cutaneous angiofibromas in pediatric subjects with tuberous sclerosis complex (TSC).

Detailed description

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study designed to assess the safety and efficacy of topically-applied sirolimus for the treatment of cutaneous angiofibromas in pediatric subjects with TSC. Approximately 45 subjects will be enrolled at investigational sites in the United States (US) and China, though other countries may be added in the future. Approximately 45 subjects who meet the study entry criteria will randomly be assigned in a 1:1:1 ratio to receive 1 of 3 treatments: sirolimus 0.2% ointment, sirolimus 0.4% ointment, or placebo ointment. The randomization is stratified by site. Subjects, or a parent/guardian, will apply the study medication topically to the cutaneous angiofibromas on the face once daily at night before going to bed for 12 weeks. Subjects who complete the double-blind phase of the study, with an overall compliance rate \>80% as determined by the dosing diary, will be offered entry into an open-label period for an additional 12 weeks. The maximum study duration for each subject will be approximately 30 weeks and includes a screening period of up to 4 weeks, a blinded treatment period of 12 weeks, optional open-label period of 12 weeks, and a follow-up period of 2 weeks. An interim analysis will be performed when all subjects have completed the double-blind phase (Visit 5 - Week 12). The data will be unblinded to assess for efficacy and results reported.

Conditions

• Angiofibroma of Face
• Tuberous Sclerosis

Interventions

Type	Name	Description
DRUG	Sirolimus 0.2%	Ointment for topical administration hs x 12 weeks
DRUG	Sirolimus 0.4%	Ointment for topical administration hs x 12 weeks
DRUG	Placebo ointment	Placebo ointment comparator for topical administration hs x 12 weeks

Timeline

• Start date: 2017-04-12
• Primary completion: 2023-03-24
• Completion: 2023-03-24
• First posted: 2017-12-06
• Last updated: 2025-12-01
• Results posted: 2025-12-01

Locations

8 sites across 2 countries: United States, China

Regulatory

• FDA-regulated drug study