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TerminatedNCT03363763Updated Dec 1, 2025Results posted

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

A Phase 2 interventional study of Sirolimus 0.2% and Sirolimus 0.4% in Angiofibroma of Face and Tuberous Sclerosis, sponsored by Aucta Pharmaceuticals, Inc. Terminated at 8 sites in 2 countries. Open to participants aged 2 Years to 21 Years. Per ClinicalTrials.gov, last updated 2025-12-01.

Sponsored by Aucta Pharmaceuticals, Inc · Phase 2, Interventional, and Treatment

Why this study was terminated
Terminated (The clinical trial has ended prematurely due to low patient recruitment)

From the registry’s dates

  • Registered 7 months after the study started (first participant enrolled Apr 2017, registered Dec 2017).
Phase
Phase 2
Study type
Interventional
Enrollment
24
Allocation
Randomized
Ages
2 Years to 21 Years
Sex
All
01

Study 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 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.

02

Conditions studied

  • Angiofibroma of Face
  • Tuberous Sclerosis

Keywords

  • rash
  • fibroma
  • skin
  • facial
  • face
  • bumps
  • redness
  • erythema
  • lesions
  • papules
  • blood vessel
  • cheeks
03

In context

Tuberous Sclerosis

115 studies on the registry are indexed under Tuberous Sclerosis; 27 are open to participants now.

This study's enrollment of 24 is below the median of 52 across 73 interventional studies indexed under Tuberous Sclerosis.

Browse Tuberous Sclerosis studies →

Lead sponsor

Aucta Pharmaceuticals, Inc is the lead sponsor of 2 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Generally healthy males or non-pregnant females aged 2 to 21 years, inclusive, at the time of screening.
  2. Diagnosis of TSC with visible facial angiofibromas of at least grade 3 up to grade 5, inclusive, based on the IGA.
  3. Subjects with 3 or more isolated, of facial angiofibroma, with color grading score ≥2 for each of the 3 lesions.

  4. Females of 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 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.
    • 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.
  5. The subject and/or their parent or guardian must be willing and able to provide written /assent.
  6. Willing and able to comply with all trial requirements.
  7. 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.
  8. Subjects should be in good general health based on the subject's medical history, physical exam, and impression of the study doctor.

Exclusion criteria

Exclusion Criteria:

  1. 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.
  2. 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.)
  3. Is currently receiving any form of therapy or has previously experienced significant immune dysfunction.
  4. Has a history of sensitivity to any component of the product.
  5. Is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
  6. 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.
  7. Has facial hair (e.g., beard, sideburns, mustache) that could interfere with study assessments.
  8. Has had laser surgery or cryotherapy to facial angiofibromas within 6 months preceding study entry.
  9. 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).
  10. Has participated in another clinical trial or received an investigational product within 3 months prior to screening.
Terms in these criteria, in plain language(6)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
24 participants (actual)

Study arms

  • Active comparator
    Sirolimus 0.2%

    Sirolimus 0.2% ointment applied topically hs x 12 weeks

    Drug: Sirolimus 0.2%

  • Active comparator
    Sirolimus 0.4%

    Sirolimus 0.4% ointment applied topically hs x 12 weeks

    Drug: Sirolimus 0.4%

  • Placebo comparator
    Placebo

    Placebo ointment applied topically hs x 12 weeks

    Drug: Placebo ointment

Interventions

  • DrugSirolimus 0.2%

    Ointment for topical administration hs x 12 weeks

    Also known as: Rapamune, rapamycin, mTOR inhibitor

  • DrugSirolimus 0.4%

    Ointment for topical administration hs x 12 weeks

    Also known as: Rapamune, rapamycin, mTOR inhibitor

  • DrugPlacebo ointment

    Placebo ointment comparator for topical administration hs x 12 weeks

    Also known as: Placebo

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Double-blind phase and Open-label phase Weeks 4 and 12

  2. 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)

    Time frame: Double-blind phase and Open-label phase Weeks 4 and 12

Secondary outcomes

  1. 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.

    Time frame: Double-blind phase Weeks 4 and 12 and Open-label phase Week 12

  2. 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.

    Time frame: Baseline, Double blind phase weeks 4 and 12 and Open-label week 12

  3. 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 \[0 = no lesion, 1 = \<25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = \> 75 lesions\]

    Time frame: Double blind phase Weeks 4 and 12 and Open-label phase Week 12

  4. 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)

    Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12

  5. 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).

    Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12

  6. 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.

    Time frame: Double blind phase weeks 4 and 12 and open-label phase week 12

  7. 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)

    Time frame: Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12

  8. 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)

    Time frame: Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12

07

Results

Posted Dec 1, 2025

Participant flow

Study subjects were enrolled at 3-5 investigational sites in the United States.

Double-Blind Phase
Participant flow — Double-Blind Phase
MilestoneSirolimus 0.2%Sirolimus 0.4%Placebo
Started789
Completed789
Not completed000
Open-label Phase
Participant flow — Open-label Phase
MilestoneSirolimus 0.2%Sirolimus 0.4%Placebo
Started789
Completed667
Not completed122

Outcome measures

PrimaryThe 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.

Time frame:
Double-blind phase and Open-label phase Weeks 4 and 12
Reported as:
Count of participants · Participants
The Percentage of Participants Achieved at Least 2-grade Improvement
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
Primary Endpoint (>=2 point improvement) Week 12 — No (N)669
Primary Endpoint (>=2 point improvement) Week 12 — Yes (Y)120
Primary Endpoint (>=2 point improvement) Week 4 — No (N)689
Primary Endpoint (>=2 point improvement) Week 4 — Yes (Y)100
Primary Endpoint (>/=2 point improvement) (Open-label Week 4) — No (N)657
Primary Endpoint (>/=2 point improvement) (Open-label Week 4) — Yes (Y)112
Primary Endpoint (>=2 point improvement) (Open-label Week 12) — No (N)453
Primary Endpoint (>=2 point improvement) (Open-label Week 12) — Yes (Y)214
PrimaryThe 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)

Time frame:
Double-blind phase and Open-label phase Weeks 4 and 12
Reported as:
Count of participants · Participants
The Change in Baseline in Investigator's Global Assessment (IGA) by Visit
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
Change from Baseline in IGA by Visit: Week 4 — 0 = No change from baseline248
Change from Baseline in IGA by Visit: Week 4 — -1 = 1- point improvement from baseline441
Change from Baseline in IGA by Visit: Week 4 — -2 = 2-point improvement from baseline100
Change from Baseline in IGA by Visit: Week 4 — -3 = 3-point improvement from baseline000
Change from Baseline in IGA by Visit: Week 12 — 0 = No change from baseline138
Change from Baseline in IGA by Visit: Week 12 — -1 = 1- point improvement from baseline531
Change from Baseline in IGA by Visit: Week 12 — -2 = 2-point improvement from baseline120
Change from Baseline in IGA by Visit: Week 12 — -3 = 3-point improvement from baseline000
Change from Baseline in IGA by Visit: Open-label week 4 — 0 = No change from baseline121
Change from Baseline in IGA by Visit: Open-label week 4 — -1 = 1- point improvement from baseline536
Change from Baseline in IGA by Visit: Open-label week 4 — -2 = 2-point improvement from baseline112
Change from Baseline in IGA by Visit: Open-label week 4 — -3 = 3-point improvement from baseline000
Change from Baseline in IGA by visit: Open-label week 12 — 0 = No change from baseline020
Change from Baseline in IGA by visit: Open-label week 12 — -1 = 1- point improvement from baseline433
Change from Baseline in IGA by visit: Open-label week 12 — -2 = 2-point improvement from baseline113
Change from Baseline in IGA by visit: Open-label week 12 — -3 = 3-point improvement from baseline101
SecondaryThe 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.

Time frame:
Double-blind phase Weeks 4 and 12 and Open-label phase Week 12
Reported as:
Count of participants · Participants
The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score.
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
>=30% Improvement in FASI Score Week 4 — No (N)569
>=30% Improvement in FASI Score Week 4 — Yes (Y)220
>=30% Improvement in FASI Score Week 12 — No (N)559
>=30% Improvement in FASI Score Week 12 — Yes (Y)230
>= 30% Improvement in FASI Score: Open-label week 12 — No (N)353
>= 30% Improvement in FASI Score: Open-label week 12 — Yes (Y)213
SecondaryFacial 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.

Time frame:
Baseline, Double blind phase weeks 4 and 12 and Open-label week 12
Reported as:
Mean · score on a scale
Facial Angifibromas Severity Index (FASI) Score
score on a scaleSirolimus 0.2%Sirolimus 0.4%Placebo
Baseline FASI Total Score6.86 ± 1.357.38 ± 1.197.89 ± 1.27
Week 4 FASI Total Score5.71 ± 1.256.5 ± 1.697.33 ± 1.32
Week 12 FASI Total Score5.57 ± 0.985.75 ± 1.397 ± 1.58
Open-label week 12 FASI Total Score5 ± 16 ± 0.635.33 ± 1.51
SecondaryThe 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 \[0 = no lesion, 1 = \<25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = \> 75 lesions\]

Time frame:
Double blind phase Weeks 4 and 12 and Open-label phase Week 12
Reported as:
Count of participants · Participants
The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
>=2 grade improvement in lesion: Week 4 — No (N)679
>=2 grade improvement in lesion: Week 4 — Yes (N)110
>=2 grade improvement in lesion: Week 12 — No (N)679
>=2 grade improvement in lesion: Week 12 — Yes (N)110
>=2 grade improvement in lesion: Open-label week 12 — No (N)454
>=2 grade improvement in lesion: Open-label week 12 — Yes (N)112
SecondaryChange 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)

Time frame:
Double blind phase weeks 4 and 12 and open-label phase week 12
Reported as:
Count of participants · Participants
Change From Baseline in Lesion Counts
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
Change from Baseline in lesion counts by visit: Week 4 — 0 = No change from baseline658
Change from Baseline in lesion counts by visit: Week 4 — -1 = 1-point improvement from baseline021
Change from Baseline in lesion counts by visit: Week 4 — -2 = 2-point improvement from baseline110
Change from Baseline in lesion counts by visit: Week 4 — -3 = 3-point improvement from baseline000
Change from Baseline in lesion counts by visit: Week 12 — 0 = No change from baseline359
Change from Baseline in lesion counts by visit: Week 12 — -1 = 1-point improvement from baseline320
Change from Baseline in lesion counts by visit: Week 12 — -2 = 2-point improvement from baseline100
Change from Baseline in lesion counts by visit: Week 12 — -3 = 3-point improvement from baseline010
Change from baseline in lesion counts by visit: Open-label week 12 — 0 = No change from baseline222
Change from baseline in lesion counts by visit: Open-label week 12 — -1 = 1-point improvement from baseline232
Change from baseline in lesion counts by visit: Open-label week 12 — -2 = 2-point improvement from baseline102
Change from baseline in lesion counts by visit: Open-label week 12 — -3 = 3-point improvement from baseline010
SecondaryThe 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).

Time frame:
Double blind phase weeks 4 and 12 and open-label phase week 12
Reported as:
Count of participants · Participants
The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation.
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
>=2 grade improvement in lesion elevation: Week 4 — No (N)689
>=2 grade improvement in lesion elevation: Week 4 — Yes (Y)100
>=2 grade improvement in lesion elevation: Week 12 — No (N)689
>=2 grade improvement in lesion elevation: Week 12 — Yes (Y)100
>=2 grade improvement in lesion elevation: Open-label week 12 — No (N)456
>=2 grade improvement in lesion elevation: Open-label week 12 — Yes (Y)110
SecondaryThe 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.

Time frame:
Double blind phase weeks 4 and 12 and open-label phase week 12
Reported as:
Count of participants · Participants
The Change From Baseline in Lesion Elevation
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
Change from Baseline in lesion elevation: Week 4 — 0 = No change from baseline448
Change from Baseline in lesion elevation: Week 4 — -1 = 1-point improvement from baseline241
Change from Baseline in lesion elevation: Week 4 — -2 = 2-point improvement from baseline100
Change from Baseline in lesion elevation: Week 12 — 0 = No change from baseline349
Change from Baseline in lesion elevation: Week 12 — -1 = 1-point improvement from baseline340
Change from Baseline in lesion elevation: Week 12 — -2 = 2-point improvement from baseline100
Change from baseline in lesion elevation: Open-label week 12 — 0 = No change from baseline233
Change from baseline in lesion elevation: Open-label week 12 — -1 = 1-point improvement from baseline223
Change from baseline in lesion elevation: Open-label week 12 — -2 = 2-point improvement from baseline110
SecondaryThe 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)

Time frame:
Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12
Reported as:
Count of participants · Participants
The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
>=2 grade improvement in self-assessment: Week 4 — No (N)679
>=2 grade improvement in self-assessment: Week 4 — Yes (Y)110
>=2 grade improvement in self-assessment: Week 12 — No (N)568
>=2 grade improvement in self-assessment: Week 12 — Yes (Y)221
>=2 grade improvement in self-assessment: open-label week 4 — No (N)528
>=2 grade improvement in self-assessment: open-label week 4 — Yes (Y)241
>=2 grade improvement in self-assessment: open-label week 12 — No (N)334
>=2 grade improvement in self-assessment: open-label week 12 — Yes (Y)333
SecondaryChange 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)

Time frame:
Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12
Reported as:
Count of participants · Participants
Change From Baseline in Self-Assessment
ParticipantsSirolimus 0.2%Sirolimus 0.4%Placebo
Change from baseline in self-assessment: Week 4 — 1 = 1-point worsening from baseline000
Change from baseline in self-assessment: Week 4 — 0 = No change from baseline338
Change from baseline in self-assessment: Week 4 — -1 = 1-point improvement from baseline341
Change from baseline in self-assessment: Week 4 — -2 = 2-point improvement from baseline110
Change from baseline in self-assessment: Week 4 — -3 = 3-point improvement from baseline000
Change from Baseline in self-assessment: Week 12 — 1 = 1-point worsening from baseline000
Change from Baseline in self-assessment: Week 12 — 0 = No change from baseline116
Change from Baseline in self-assessment: Week 12 — -1 = 1-point improvement from baseline452
Change from Baseline in self-assessment: Week 12 — -2 = 2-point improvement from baseline121
Change from Baseline in self-assessment: Week 12 — -3 = 3-point improvement from baseline100
Change from baseline in self- assessment: open-label week 14 — 1 = 1-point worsening from baseline000
Change from baseline in self- assessment: open-label week 14 — 0 = No change from baseline123
Change from baseline in self- assessment: open-label week 14 — -1 = 1-point improvement from baseline405
Change from baseline in self- assessment: open-label week 14 — -2 = 2-point improvement from baseline231
Change from baseline in self- assessment: open-label week 14 — -3 = 3-point improvement from baseline010
Change from baseline in self-assessment: open-label week 12 — 1 = 1-point worsening from baseline001
Change from baseline in self-assessment: open-label week 12 — 0 = No change from baseline011
Change from baseline in self-assessment: open-label week 12 — -1 = 1-point improvement from baseline322
Change from baseline in self-assessment: open-label week 12 — -2 = 2-point improvement from baseline233
Change from baseline in self-assessment: open-label week 12 — -3 = 3-point improvement from baseline100

Adverse events

Collected over 30 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Double-Blind: Sirolimus 0.2%0/7 (0%)0/7 (0%)6/7 (85.7%)
Double-Blind: Sirolimus 0.4%0/8 (0%)0/8 (0%)5/8 (62.5%)
Double-Blind: Placebo0/9 (0%)1/9 (11.1%)4/9 (44.4%)
Open-label: Sirolimus 0.2%0/7 (0%)1/7 (14.3%)1/7 (14.3%)
Open-label: Sirolimus 0.4%0/7 (0%)1/7 (14.3%)5/7 (71.4%)
Open-label: Placebo0/9 (0%)0/9 (0%)4/9 (44.4%)
Most frequent serious events
Most frequent serious events
EventDouble-Blind: Sirolimus 0.2%Double-Blind: Sirolimus 0.4%Double-Blind: PlaceboOpen-label: Sirolimus 0.2%Open-label: Sirolimus 0.4%Open-label: Placebo
Ligament ruptureInjury, poisoning and procedural complications0/70/80/90/71/70/9
Psychotic disorder due to a general medical conditionPsychiatric disorders0/70/80/91/70/70/9
AgitationPsychiatric disorders0/70/81/90/70/70/9
Most frequent other events
Showing 10 of 28
Most frequent other events
EventDouble-Blind: Sirolimus 0.2%Double-Blind: Sirolimus 0.4%Double-Blind: PlaceboOpen-label: Sirolimus 0.2%Open-label: Sirolimus 0.4%Open-label: Placebo
AcneSkin and subcutaneous tissue disorders0/72/80/91/70/70/9
Upper respiratory tract infectionInfections and infestations1/70/80/90/71/72/9
Mouth UlcerationGastrointestinal disorders1/70/80/90/70/70/9
Application site reactionGeneral disorders1/70/80/90/70/70/9
PyrexiaGeneral disorders0/70/81/90/71/70/9
AnxietyPsychiatric disorders1/70/81/90/70/70/9
Obsessive thoughtsPsychiatric disorders1/70/80/90/70/70/9
Dermatitis acneiformSkin and subcutaneous tissue disorders1/70/80/90/70/70/9
RashSkin and subcutaneous tissue disorders1/70/80/90/70/71/9
Skin irritationSkin and subcutaneous tissue disorders1/70/81/90/70/70/9

Baseline characteristics

\[Not Specified\]

Age, Continuous
Age, Continuous(Years)Arm 1Arm 2Arm 3Total
Mean12.1 ± 2.7313.1 ± 4.6710.9 ± 3.4412.0 ± 3.69
Sex: Female, Male
Sex: Female, Male(Participants)Arm 1Arm 2Arm 3Total
Female46717
Male3227
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Arm 1Arm 2Arm 3Total
Hispanic or Latino1225
Not Hispanic or Latino66719
Unknown or Not Reported0000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Arm 1Arm 2Arm 3Total
American Indian or Alaska Native0000
Asian2327
Native Hawaiian or Other Pacific Islander0000
Black or African American0000
White55616
More than one race0000
Unknown or Not Reported0011
Height (cm)
Height (cm)(cm)Arm 1Arm 2Arm 3Total
Mean154.8 ± 11.65144.9 ± 23.04141.2 ± 16.39146.6 ± 18.02
Weight (kg)
Weight (kg)(kg)Arm 1Arm 2Arm 3Total
Mean50.3 ± 20.0841.3 ± 18.2037.0 ± 9.3742.3 ± 16.35
BMI (kg/m^2)
BMI (kg/m^2)(kg/m^2)Arm 1Arm 2Arm 3Total
Mean20.6 ± 6.5218.7 ± 3.9218.7 ± 2.8519.2 ± 4.46
08

Study locations

8 sites
  • Translational Genomics Research
    Phoenix, Arizona 85004, United States
  • Children's Hospital of Los Angeles, Division of Neurology
    Los Angeles, California 90027, United States
  • Children's Clinical Research Organization, Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Children's Healthcare of Atlanta
    Atlanta, Georgia 30329, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • LeBonheur Children's Hospital
    Memphis, Tennessee 38103, United States
  • Children's Hospital of Fudan University
    Shanghai, 201102, China
09

References and documents

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 documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 1, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03363763
Lead sponsor
Aucta Pharmaceuticals, Inc
Responsible party
Sponsor
First posted
Dec 6, 2017
Start date
Apr 12, 2017
Primary completion
Mar 24, 2023
Completion
Mar 24, 2023
Results posted
Dec 1, 2025
Last update
Dec 1, 2025

Study contacts

Shoufeng Li, Ph.D
study director · Aucta Pharmaceuticals, Inc

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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