Angiofibroma of FacePhase 2NCT03363763

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

A Phase 2 study testing Sirolimus 0.2% and Sirolimus 0.4% in people with Angiofibroma of Face and Tuberous Sclerosis, run by Aucta Pharmaceuticals, Inc at 8 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial 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). Read more about this study

Who is it for?: Ages 2 to 21 years
Conditions the study is for: Angiofibroma of Face, Tuberous Sclerosis
What is being tested?: Sirolimus 0.2% and Sirolimus 0.4%
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 24 participants
Where: 8 sitesin 2 countries
Study dates: 12 Apr 2017to 24 Mar 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Generally healthy males or non-pregnant females aged 2 to 21 years, inclusive, at the time of screening.
Diagnosis of TSC with visible facial angiofibromas of at least grade 3 up to grade 5, inclusive, based on the IGA.
Subjects with 3 or more isolated, measurable lesions of facial angiofibroma, with color grading score ≥2 for each of the 3 lesions.
Females of childbearing potential must have a negative urine pregnancy test (or a negative serum pregnancy test if a urine pregnancy test cannot be obtained) (For China, different pregnancy test would be followed) and if sexually active or become sexually active during the study, must agree to use an effective form of birth control for the duration of the study. Females using oral contraceptives must also use a barrier method of contraception during the study. Sexually active male subjects and/or their female partners should also use appropriate contraception.
Effective contraception is defined as follows:
Oral/implant/injectable/transdermal/estrogenic vaginal ring contraceptives, intrauterine device, condom with spermicide, diaphragm with spermicide.

Show all 11Show fewer

Abstinence or partner's vasectomy are acceptable if the female agrees to implement one of the other acceptable methods of birth control if her partner changes.
The subject and/or their parent or guardian must be willing and able to provide written informed consent/assent.
Willing and able to comply with all trial requirements.
Subject or parent/guardian must be able to complete the subject self-assessment survey and subject diary in English or another language into which the documents have been officially translated.
Subjects should be in good general health based on the subject's medical history, physical exam, and impression of the study doctor.

Who cannot join

Has any chronic or acute medical condition, that in the opinion of the investigator, may pose a risk to the safety of the subject during the trial period, or may interfere with the assessment of safety or efficacy in this trial.
Has received oral therapy or topical therapy of an mTOR inhibitor (sirolimus, temsirolimus, or everolimus) within 1 month of Baseline or other dermatological treatment to facial angiofibromas within 1 month of baseline. (Sunscreen is expected to be used in this patient population and is not considered treatment.)
Is currently receiving any form of immunosuppression therapy or has previously experienced significant immune dysfunction.
Has a history of sensitivity to any component of the investigational product.
Is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
Has other dermatologic conditions, pigmentation, scarring, pigmented lesions or sunburn in the treatment area that would preclude or prevent adequate assessment of changes to their facial angiofibromas.

Show all 10Show fewer

Has facial hair (e.g., beard, sideburns, mustache) that could interfere with study assessments.
Has had laser surgery or cryotherapy to facial angiofibromas within 6 months preceding study entry.
Requires the use of any concomitant medication that, in the investigator's opinion, has the potential to cause an adverse effect when given with the investigational product or will interfere with the interpretation of the study results (see Section 16.1 Appendix 1 for Potential Drug Interactions).
Has participated in another clinical trial or received an investigational product within 3 months prior to screening.

Trial locations and contacts

8 sites in 2 countries.

Country

City, postcode or hospital

8 sites in 2 countries

United States7 sites

Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Children's Clinical Research Organization, Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Children's Hospital of Los Angeles, Division of Neurology
Los Angeles, California, 90027
LeBonheur Children's Hospital
Memphis, Tennessee, 38103
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Translational Genomics Research
Phoenix, Arizona, 85004

Other trials in United States

China1 site

Children's Hospital of Fudan University
Shanghai, 201102

Other trials in China

About this study

Official title: 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

Read the 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
Being tested: Sirolimus 0.2%, Sirolimus 0.4%, Placebo ointment
Keywords: rash, fibroma, skin, facial, face, bumps, redness, erythema, lesions, papules, blood vessel, cheeks
Medical subject headings: Tuberous Sclerosis, Exanthema, Fibroma, Facies, Erythema

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Sirolimus 0.2%Drug
Also called Rapamune, rapamycin, mTOR inhibitor
Ointment for topical administration hs x 12 weeks
Sirolimus 0.4%Drug
Also called Rapamune, rapamycin, mTOR inhibitor
Ointment for topical administration hs x 12 weeks
Placebo ointmentDrug
Also called Placebo
Placebo ointment comparator for topical administration hs x 12 weeks

Study groups in NCT03363763
Study group	Role	What they receive
Sirolimus 0.2%	Active comparator	Sirolimus 0.2% ointment applied topically hs x 12 weeks Receives: Drug: Sirolimus 0.2%
Sirolimus 0.4%	Active comparator	Sirolimus 0.4% ointment applied topically hs x 12 weeks Receives: Drug: Sirolimus 0.4%
Placebo	Placebo comparator	Placebo ointment applied topically hs x 12 weeks Receives: Drug: Placebo ointment

What is being measured

The primary outcome is the main question the study is designed to answer.

The Percentage of Participants Achieved at Least 2-grade Improvement
The Investigator's Global Assessment (IGA) was recorded on a 5-point scale, 0 (minimum) to 5 (maximum) [0 = Clear skin with no signs of erythema and no disease related lesions, 1 = Slight redness with few disease related lesions, 2 = Greater than Grade 1; definite redness with scattered, some disease related lesions, 3 = Greater than Grade 2; marked redness, concentrated, many disease related lesions, 4 = Greater than Grade 3; very bright redness, confluent, highly concentrated disease related lesions, 5= Greater than Grade 4; fiery redness, very extensive disease related lesions covering very large area of the face]. A higher score indicates a more severe, worse outcome.
Measured: Double-blind phase and Open-label phase Weeks 4 and 12
The Change in Baseline in Investigator's Global Assessment (IGA) by Visit
The Investigator's Global Assessment (IGA) was recorded on a 5-point scale, 0 (minimum) to 5 (maximum) [0 = Clear skin with no signs of erythema and no disease related lesions, 1 = Slight redness with few disease related lesions, 2 = Greater than Grade 1; definite redness with scattered, some disease related lesions, 3 = Greater than Grade 2; marked redness, concentrated, many disease related lesions, 4 = Greater than Grade 3; very bright redness, confluent, highly concentrated disease related lesions, 5= Greater than Grade 4; fiery redness, very extensive disease related lesions covering very large area of the face]. A higher score indicates a more severe, worse outcome.
Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement)
Measured: Double-blind phase and Open-label phase Weeks 4 and 12

Secondary outcomes (8)

The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score.
The Facial Angiofibromas Severity Index (FASI) is derived from the measurements of erythema, average size, and lesion extension. The FASI score is the sum of scores of Erythema, Size, and Extension. Erythema was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = clear skin with no signs of erythema, 1 = almost clear; slight redness, 3= moderate erythema; marked redness, 4= severe erythema; very bright redness]. Size was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = no lesions, 1= few lesions, average size </= 2mm, 2 = scattered, some lesions, average lesion size >2-to </= 5 mm, 3 = concentrated, many lesions, average lesion size > 5mmg to </= 10mm, 4 = confluent, highly concentrated lesions]. Extension was recorded on a grade scale of 0 (minimum), 2, and 3 (maximum) [0 = no lesions, 2 = </= 50% of the cheek surface, 3 = >50% of the cheek surface]. A higher score for Erythema, Size, and Extension means a more severe, worse outcome.
Measured: Double-blind phase Weeks 4 and 12 and Open-label phase Week 12
Facial Angifibromas Severity Index (FASI) Score
The Facial Angiofibromas Severity Index (FASI) is derived from the measurements of erythema, average size, and lesion extension. The FASI score is the sum of scores of Erythema, Size, and Extension. Erythema was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = clear skin with no signs of erythema, 1 = almost clear; slight redness, 3= moderate erythema; marked redness, 4= severe erythema; very bright redness]. Size was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = no lesions, 1= few lesions, average size </= 2mm, 2 = scattered, some lesions, average lesion size >2-to </= 5 mm, 3 = concentrated, many lesions, average lesion size > 5mmg to </= 10mm, 4 = confluent, highly concentrated lesions]. Extension was recorded on a grade scale of 0 (minimum), 2, and 3 (maximum) [0 = no lesions, 2 = </= 50% of the cheek surface, 3 = >50% of the cheek surface]. A higher score for Erythema, Size, and Extension means a more severe, worse outcome.
Measured: Baseline, Double blind phase weeks 4 and 12 and Open-label week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit
Lesion counts are measured based on categories 0 to 4 [0 = no lesion, 1 = <25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = > 75 lesions]
Measured: Double blind phase Weeks 4 and 12 and Open-label phase Week 12
Change From Baseline in Lesion Counts
Lesion counts are measured based on categories 0 to 4 (0 = no lesion, 1 = <25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = > 75 lesions) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement)
Measured: Double blind phase weeks 4 and 12 and open-label phase week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation.
The degree of lesion elevation is assessed on categories 0 to 4 (0 = no elevation over normal skin, 1 = possible but difficult to ascertain whether there is slight elevation above normal skin, 2 = slight but definite elevation, 3 = moderate elevation, 4 = marked elevation).
Measured: Double blind phase weeks 4 and 12 and open-label phase week 12
The Change From Baseline in Lesion Elevation
The degree of lesion elevation is assessed on categories 0 to 4 (0 = no elevation over normal skin, 1 = possible but difficult to ascertain whether there is slight elevation above normal skin, 2 = slight but definite elevation, 3 = moderate elevation, 4 = marked elevation) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement) One participant with baseline elevation grade of 1 improved to grade 0 at week 4. This participant is counted as improvement in lesion elevation. This participant was back to grade 1 at week 12 and open-label week 12.
Measured: Double blind phase weeks 4 and 12 and open-label phase week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey
Subject self- assessment survey was based on categories 0 to 4 (0 = no redness and no disease related lesions, 1 = very mild redness with few very small bumps, 2 = mild redness with many small and medium sized bumps, 3 = moderate redness with many small and medium sized bumps, 4 = severe redness with numerous small, medium, and large sized bumps)
Measured: Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12
Change From Baseline in Self-Assessment
Subject self- assessment survey was based on categories 0 to 4 (0 = no redness and no disease related lesions, 1 = very mild redness with few very small bumps, 2 = mild redness with many small and medium sized bumps, 3 = moderate redness with many small and medium sized bumps, 4 = severe redness with numerous small, medium, and large sized bumps) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement)
Measured: Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

12 Apr 201724 Mar 2023

First posted6 Dec 2017
Study start12 Apr 2017
Primary completion24 Mar 2023
Study completion24 Mar 2023

Why it stopped: Terminated (The clinical trial has ended prematurely due to low patient recruitment)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Angiofibroma of FaceStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Subjects will be randomly assigned in a 1:1:1 ratio to receive 1 of 2 treatments or placebo. The randomization is stratified by site. Subjects who complete the double-blind phase of the study with an overall compliance rate \>80% and \<120%, as determined by weight of returned study medication, will be offered entry into an open-label period for an additional 12 weeks.
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 24 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03363763
Sponsor's protocol ID: AUCTA-UAP006-PH2
Status last verified: Nov 2025
First submitted: 1 Dec 2017
Last updated: 1 Dec 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.
PubMed 22934754
Wheless JW, Almoazen H. A novel topical rapamycin cream for the treatment of facial angiofibromas in tuberous sclerosis complex. J Child Neurol. 2013 Jul;28(7):933-6. doi: 10.1177/0883073813488664. Epub 2013 May 16.
PubMed 23680945
Wataya-Kaneda M, Tanaka M, Nakamura A, Matsumoto S, Katayama I. A topical combination of rapamycin and tacrolimus for the treatment of angiofibroma due to tuberous sclerosis complex (TSC): a pilot study of nine Japanese patients with TSC of different disease severity. Br J Dermatol. 2011 Oct;165(4):912-6. doi: 10.1111/j.1365-2133.2011.10471.x.
PubMed 21692771
Tanaka M, Wataya-Kaneda M, Nakamura A, Matsumoto S, Katayama I. First left-right comparative study of topical rapamycin vs. vehicle for facial angiofibromas in patients with tuberous sclerosis complex. Br J Dermatol. 2013 Dec;169(6):1314-8. doi: 10.1111/bjd.12567.
PubMed 23909960
Rapamune (sirolimus) complete prescribing information. Wyeth Pharmaceuticals Inc. October 2009

Study at a glance

From the registry record, last updated by the sponsor on 1 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT03363763
Acronym / sponsor ID: AUCTA-UAP006-PH2
Conditions: Angiofibroma of Face, Tuberous Sclerosis
Keywords: rash, fibroma, skin, facial, face, bumps, redness, erythema
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Terminated (The clinical trial has ended prematurely due to low patient recruitment)
Enrolment: 24 participants (actual)
Lead sponsor: Aucta Pharmaceuticals, IncIndustry (pharma, biotech, devices)
Study start: 12 Apr 2017 (actual)
Primary completion: 24 Mar 2023 (actual)
Study completion: 24 Mar 2023 (actual)
First posted: 6 Dec 2017 (actual)
Results: Posted 1 Dec 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3
Model description: Subjects will be randomly assigned in a 1:1:1 ratio to receive 1 of 2 treatments or placebo. The randomization is stratified by site. Subjects who complete the double-blind phase of the study with an overall compliance rate >80% and <120%, as determined by weight of returned study medication, will be offered entry into an open-label period for an additional 12 weeks.

Arm	Type	Receives
Sirolimus 0.2%	Active comparator	Drug: Sirolimus 0.2%Sirolimus 0.2% ointment applied topically hs x 12 weeks
Sirolimus 0.4%	Active comparator	Drug: Sirolimus 0.4%Sirolimus 0.4% ointment applied topically hs x 12 weeks
Placebo	Placebo comparator	Drug: Placebo ointmentPlacebo ointment applied topically hs x 12 weeks

Who may qualify

Ages: 2 to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (11 listed)

Generally healthy males or non-pregnant females aged 2 to 21 years, inclusive, at the time of screening.
Diagnosis of TSC with visible facial angiofibromas of at least grade 3 up to grade 5, inclusive, based on the IGA.
Subjects with 3 or more isolated, measurable lesions of facial angiofibroma, with color grading score ≥2 for each of the 3 lesions.
Females of childbearing potential must have a negative urine pregnancy test (or a negative serum pregnancy test if a urine pregnancy test cannot be obtained) (For China, different pregnancy test would be followed) and if sexually active or become sexually active during the study, must agree to use an effective form of birth control for the duration of the study. Females using oral contraceptives must also use a barrier method of contraception during the study. Sexually active male subjects and/or their female partners should also use appropriate contraception.
The subject and/or their parent or guardian must be willing and able to provide written informed consent/assent.

Key exclusion criteria (10 listed)

Has any chronic or acute medical condition, that in the opinion of the investigator, may pose a risk to the safety of the subject during the trial period, or may interfere with the assessment of safety or efficacy in this trial.
Has received oral therapy or topical therapy of an mTOR inhibitor (sirolimus, temsirolimus, or everolimus) within 1 month of Baseline or other dermatological treatment to facial angiofibromas within 1 month of baseline. (Sunscreen is expected to be used in this patient population and is not considered treatment.)
Is currently receiving any form of immunosuppression therapy or has previously experienced significant immune dysfunction.
Has a history of sensitivity to any component of the investigational product.
Is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Sirolimus 0.2%Also: Rapamune, rapamycin, mTOR inhibitorOintment for topical administration hs x 12 weeks	Drug	Sirolimus 0.2%
Sirolimus 0.4%Also: Rapamune, rapamycin, mTOR inhibitorOintment for topical administration hs x 12 weeks	Drug	Sirolimus 0.4%
Placebo ointmentAlso: PlaceboPlacebo ointment comparator for topical administration hs x 12 weeks	Drug	Placebo

Primary outcome

The Percentage of Participants Achieved at Least 2-grade Improvement
Time frame: Double-blind phase and Open-label phase Weeks 4 and 12
The Investigator's Global Assessment (IGA) was recorded on a 5-point scale, 0 (minimum) to 5 (maximum) [0 = Clear skin with no signs of erythema and no disease related lesions, 1 = Slight redness with few disease related lesions, 2 = Greater than Grade 1; definite redness with scattered, some disease related lesions, 3 = Greater than Grade 2; marked redness, concentrated, many disease related lesions, 4 = Greater than Grade 3; very bright redness, confluent, highly concentrated disease related lesions, 5= Greater than Grade 4; fiery redness, very extensive disease related lesions covering very large area of the face]. A higher score indicates a more severe, worse outcome.
The Change in Baseline in Investigator's Global Assessment (IGA) by Visit
Time frame: Double-blind phase and Open-label phase Weeks 4 and 12
The Investigator's Global Assessment (IGA) was recorded on a 5-point scale, 0 (minimum) to 5 (maximum) [0 = Clear skin with no signs of erythema and no disease related lesions, 1 = Slight redness with few disease related lesions, 2 = Greater than Grade 1; definite redness with scattered, some disease related lesions, 3 = Greater than Grade 2; marked redness, concentrated, many disease related lesions, 4 = Greater than Grade 3; very bright redness, confluent, highly concentrated disease related lesions, 5= Greater than Grade 4; fiery redness, very extensive disease related lesions covering very large area of the face]. A higher score indicates a more severe, worse outcome. Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement)

8 secondary outcomes

The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score.
Time frame: Double-blind phase Weeks 4 and 12 and Open-label phase Week 12
Facial Angifibromas Severity Index (FASI) Score
Time frame: Baseline, Double blind phase weeks 4 and 12 and Open-label week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit
Time frame: Double blind phase Weeks 4 and 12 and Open-label phase Week 12
Change From Baseline in Lesion Counts
Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation.
Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12
The Change From Baseline in Lesion Elevation
Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12
The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey
Time frame: Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12
Change From Baseline in Self-Assessment
Time frame: Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12

Results and safety

Results posted to the registry on 1 Dec 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Sirolimus 0.2%	7	7
Sirolimus 0.4%	8	8
Placebo	9	9

Primary outcome: The Percentage of Participants Achieved at Least 2-grade Improvement

Time frame: Double-blind phase and Open-label phase Weeks 4 and 12. Count of participants. Unit: Participants

Measure	Sirolimus 0.2% n = 7	Sirolimus 0.4% n = 8	Placebo n = 9
Primary Endpoint (>=2 point improvement) Week 12: No (N)	6	6	9
Primary Endpoint (>=2 point improvement) Week 12: Yes (Y)	1	2	0
Primary Endpoint (>=2 point improvement) Week 4: No (N)	6	8	9
Primary Endpoint (>=2 point improvement) Week 4: Yes (Y)	1	0	0
Primary Endpoint (>/=2 point improvement) (Open-label Week 4): No (N)	6	5	7
Primary Endpoint (>/=2 point improvement) (Open-label Week 4): Yes (Y)	1	1	2
Primary Endpoint (>=2 point improvement) (Open-label Week 12): No (N)	4	5	3
Primary Endpoint (>=2 point improvement) (Open-label Week 12): Yes (Y)	2	1	4

Population analysed: In the double-blind phase all randomized participants received study treatment and completed week 12 Investigator Global Assessment (IGA). In open-label phase, total of 19 participants completed week 24 (Open-label week 12) Investigator Global Assessment (IGA).

Primary outcome: The Change in Baseline in Investigator's Global Assessment (IGA) by Visit

Time frame: Double-blind phase and Open-label phase Weeks 4 and 12. Count of participants. Unit: Participants

Measure	Sirolimus 0.2% n = 7	Sirolimus 0.4% n = 8	Placebo n = 9
Change from Baseline in IGA by Visit: Week 4: 0 = No change from baseline	2	4	8
Change from Baseline in IGA by Visit: Week 4: -1 = 1- point improvement from baseline	4	4	1
Change from Baseline in IGA by Visit: Week 4: -2 = 2-point improvement from baseline	1	0	0
Change from Baseline in IGA by Visit: Week 4: -3 = 3-point improvement from baseline	0	0	0
Change from Baseline in IGA by Visit: Week 12: 0 = No change from baseline	1	3	8
Change from Baseline in IGA by Visit: Week 12: -1 = 1- point improvement from baseline	5	3	1
Change from Baseline in IGA by Visit: Week 12: -2 = 2-point improvement from baseline	1	2	0
Change from Baseline in IGA by Visit: Week 12: -3 = 3-point improvement from baseline	0	0	0

Population analysed: In the double-blind phase, all randomized participants received study treatment and completed week 12 Investigator Global Assessment (IGA). In open-label phase, total of 19 participants completed week 24 (Open-label week 12) Investigator Global Assessment (IGA).

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: 30 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Double-Blind: Sirolimus 0.2%	0 of 7 (0%)	6 of 7 (85.7%)	0 of 7
Double-Blind: Sirolimus 0.4%	0 of 8 (0%)	5 of 8 (62.5%)	0 of 8
Double-Blind: Placebo	1 of 9 (11.1%)	4 of 9 (44.4%)	0 of 9
Open-label: Sirolimus 0.2%	1 of 7 (14.3%)	1 of 7 (14.3%)	0 of 7
Open-label: Sirolimus 0.4%	1 of 7 (14.3%)	5 of 7 (71.4%)	0 of 7
Open-label: Placebo	0 of 9 (0%)	4 of 9 (44.4%)	0 of 9

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 22934754Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.
Background: PubMed 23680945Wheless JW, Almoazen H. A novel topical rapamycin cream for the treatment of facial angiofibromas in tuberous sclerosis complex. J Child Neurol. 2013 Jul;28(7):933-6. doi: 10.1177/0883073813488664. Epub 2013 May 16.
Background: PubMed 21692771Wataya-Kaneda M, Tanaka M, Nakamura A, Matsumoto S, Katayama I. A topical combination of rapamycin and tacrolimus for the treatment of angiofibroma due to tuberous sclerosis complex (TSC): a pilot study of nine Japanese patients with TSC of different disease severity. Br J Dermatol. 2011 Oct;165(4):912-6. doi: 10.1111/j.1365-2133.2011.10471.x.
Background: PubMed 23909960Tanaka M, Wataya-Kaneda M, Nakamura A, Matsumoto S, Katayama I. First left-right comparative study of topical rapamycin vs. vehicle for facial angiofibromas in patients with tuberous sclerosis complex. Br J Dermatol. 2013 Dec;169(6):1314-8. doi: 10.1111/bjd.12567.
BackgroundRapamune (sirolimus) complete prescribing information. Wyeth Pharmaceuticals Inc. October 2009
Individual participant dataSharing planned: No

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Shoufeng Li, Ph.D study director

Aucta Pharmaceuticals, Inc

8 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sirolimus 0.2% and Sirolimus 0.4% in people with Angiofibroma of Face and Tuberous Sclerosis, run by Aucta Pharmaceuticals, Inc at 8 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 1 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States, China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The Percentage of Participants Achieved at Least 2-grade Improvement (time frame: Double-blind phase and Open-label phase Weeks 4 and 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03363763, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Dec 2025; AzTrials last refreshed its copy on 7 Nov 2026.