Angiofibroma of Face, Tuberous Sclerosis
Conditions
Keywords
rash, fibroma, skin, facial, face, bumps, redness, erythema, lesions, papules, blood vessel, cheeks
Brief 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.
Interventions
DRUGSirolimus 0.2%
Ointment for topical administration hs x 12 weeks
DRUGSirolimus 0.4%
Ointment for topical administration hs x 12 weeks
DRUGPlacebo ointment
Placebo ointment comparator for topical administration hs x 12 weeks
Sponsors
Aucta Pharmaceuticals, Inc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention 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.
Eligibility
Sex/Gender
ALL
Age
2 Years to 21 Years
Healthy volunteers
No
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, measurable lesions of facial angiofibroma, with color grading score ≥2 for each of the 3 lesions. 4. 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. * 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 informed consent/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
9. 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. 10. 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.) 11. Is currently receiving any form of immunosuppression therapy or has previously experienced significant immune dysfunction. 12. Has a history of sensitivity to any component of the investigational product. 13. Is pregnant, plans to become pregnant during the course of the study, or is breastfeeding. 14. 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. 15. Has facial hair (e.g., beard, sideburns, mustache) that could interfere with study assessments. 16. Has had laser surgery or cryotherapy to facial angiofibromas within 6 months preceding study entry. 17. 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). 18. Has participated in another clinical trial or received an investigational product within 3 months prior to screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Percentage of Participants Achieved at Least 2-grade Improvement | 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 | 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | Double blind phase Weeks 4 and 12 and Open-label phase Week 12 | Lesion counts are measured based on categories 0 to 4 \[0 = no lesion, 1 = \<25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = \> 75 lesions\] |
| Change From Baseline in Lesion Counts | Double blind phase weeks 4 and 12 and open-label phase week 12 | 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) |
| The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | Double blind phase weeks 4 and 12 and open-label phase week 12 | 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). |
| The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | Double-blind phase Weeks 4 and 12 and Open-label phase Week 12 | 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. |
| The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12 | 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) |
| Change From Baseline in Self-Assessment | Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12 | 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) |
| The Change From Baseline in Lesion Elevation | Double blind phase weeks 4 and 12 and open-label phase week 12 | 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. |
| Facial Angifibromas Severity Index (FASI) Score | Baseline, Double blind phase weeks 4 and 12 and Open-label week 12 | 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. |
Countries
China, United States
Participant flow
Recruitment details
Study subjects were enrolled at 3-5 investigational sites in the United States.
Pre-assignment details
At the time of study closure, a total of 28 participants were screened and 24 were randomized and received treatment. This study was early terminated due to another company receiving exclusivity.
Participants by arm
| Arm | Count |
|---|---|
| Arm 1 Sirolimus 0.2% ointment applied topically once daily at bedtime for 12 weeks | 7 |
| Arm 2 Sirolimus 0.4% ointment applied topically once daily at bedtime for 12 weeks | 8 |
| Arm 3 Placebo ointment applied topically once daily at bedtime for 12 weeks | 9 |
| Total | 24 |
Baseline characteristics
| Characteristic | Arm 1 | Total | Arm 3 | Arm 2 |
|---|---|---|---|---|
| Age, Continuous | 12.1 Years STANDARD_DEVIATION 2.73 | 12.0 Years STANDARD_DEVIATION 3.69 | 10.9 Years STANDARD_DEVIATION 3.44 | 13.1 Years STANDARD_DEVIATION 4.67 |
| BMI (kg/m^2) | 20.6 kg/m^2 STANDARD_DEVIATION 6.52 | 19.2 kg/m^2 STANDARD_DEVIATION 4.46 | 18.7 kg/m^2 STANDARD_DEVIATION 2.85 | 18.7 kg/m^2 STANDARD_DEVIATION 3.92 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 5 Participants | 2 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 19 Participants | 7 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Height (cm) | 154.8 cm STANDARD_DEVIATION 11.65 | 146.6 cm STANDARD_DEVIATION 18.02 | 141.2 cm STANDARD_DEVIATION 16.39 | 144.9 cm STANDARD_DEVIATION 23.04 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 7 Participants | 2 Participants | 3 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) White | 5 Participants | 16 Participants | 6 Participants | 5 Participants |
| Sex: Female, Male Female | 4 Participants | 17 Participants | 7 Participants | 6 Participants |
| Sex: Female, Male Male | 3 Participants | 7 Participants | 2 Participants | 2 Participants |
| Weight (kg) | 50.3 kg STANDARD_DEVIATION 20.08 | 42.3 kg STANDARD_DEVIATION 16.35 | 37.0 kg STANDARD_DEVIATION 9.37 | 41.3 kg STANDARD_DEVIATION 18.2 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk |
|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 8 | 0 / 9 | 0 / 7 | 0 / 7 | 0 / 9 |
| other Total, other adverse events | 6 / 7 | 5 / 8 | 4 / 9 | 1 / 7 | 5 / 7 | 4 / 9 |
| serious Total, serious adverse events | 0 / 7 | 0 / 8 | 1 / 9 | 1 / 7 | 1 / 7 | 0 / 9 |
Outcome results
Primary
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
Population: 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).
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -1 = 1- point improvement from baseline | 5 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | 0 = No change from baseline | 0 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -1 = 1- point improvement from baseline | 4 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -1 = 1- point improvement from baseline | 5 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -1 = 1- point improvement from baseline | 4 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | 0 = No change from baseline | 4 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -1 = 1- point improvement from baseline | 4 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -1 = 1- point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -2 = 2-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -1 = 1- point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -1 = 1- point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -1 = 1- point improvement from baseline | 6 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -1 = 1- point improvement from baseline | 1 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 3 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -2 = 2-point improvement from baseline | 2 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | 0 = No change from baseline | 8 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -1 = 1- point improvement from baseline | 1 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | -1 = 1- point improvement from baseline | 3 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | 0 = No change from baseline | 8 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by visit: Open-label week 12 | 0 = No change from baseline | 0 Participants |
| Placebo | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Change from Baseline in IGA by Visit: Open-label week 4 | 0 = No change from baseline | 1 Participants |
Primary
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
Population: 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).
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | No (N) | 4 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | Yes (Y) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | Yes (Y) | 2 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | Yes (Y) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | Yes (Y) | 0 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | No (N) | 6 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | No (N) | 8 Participants |
| Sirolimus 0.4% | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | Yes (Y) | 2 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | Yes (Y) | 4 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | No (N) | 7 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>/=2 point improvement) (Open-label Week 4) | Yes (Y) | 2 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | No (N) | 9 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 12 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | No (N) | 9 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) Week 4 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Participants Achieved at Least 2-grade Improvement | Primary Endpoint (>=2 point improvement) (Open-label Week 12) | No (N) | 3 Participants |
Secondary
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
Population: \[Not Specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | 0 = No change from baseline | 6 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -1 = 1-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -1 = 1-point improvement from baseline | 3 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | 0 = No change from baseline | 5 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | 0 = No change from baseline | 5 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -1 = 1-point improvement from baseline | 3 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | 0 = No change from baseline | 9 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -1 = 1-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -2 = 2-point improvement from baseline | 2 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | 0 = No change from baseline | 8 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -1 = 1-point improvement from baseline | 1 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | 0 = No change from baseline | 2 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from Baseline in lesion counts by visit: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Lesion Counts | Change from baseline in lesion counts by visit: Open-label week 12 | -3 = 3-point improvement from baseline | 0 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -2 = 2-point improvement from baseline | 2 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -1 = 1-point improvement from baseline | 4 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -1 = 1-point improvement from baseline | 4 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -1 = 1-point improvement from baseline | 3 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -1 = 1-point improvement from baseline | 3 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -2 = 2-point improvement from baseline | 2 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 0 = No change from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.2% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -1 = 1-point improvement from baseline | 4 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -1 = 1-point improvement from baseline | 5 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -2 = 2-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -1 = 1-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -2 = 2-point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -3 = 3-point improvement from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 1 = 1-point worsening from baseline | 0 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 0 = No change from baseline | 1 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -2 = 2-point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 1 = 1-point worsening from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -2 = 2-point improvement from baseline | 1 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 0 = No change from baseline | 6 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | 1 = 1-point worsening from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -2 = 2-point improvement from baseline | 3 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 1 = 1-point worsening from baseline | 1 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | 0 = No change from baseline | 1 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -3 = 3-point improvement from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | -1 = 1-point improvement from baseline | 1 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 1 = 1-point worsening from baseline | 0 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: Week 4 | 0 = No change from baseline | 8 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | 0 = No change from baseline | 3 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from Baseline in self-assessment: Week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self-assessment: open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Placebo | Change From Baseline in Self-Assessment | Change from baseline in self- assessment: open-label week 14 | -1 = 1-point improvement from baseline | 5 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Sirolimus 0.2% | Facial Angifibromas Severity Index (FASI) Score | Baseline FASI Total Score | 6.86 score on a scale | Standard Deviation 1.35 |
| Sirolimus 0.2% | Facial Angifibromas Severity Index (FASI) Score | Week 4 FASI Total Score | 5.71 score on a scale | Standard Deviation 1.25 |
| Sirolimus 0.2% | Facial Angifibromas Severity Index (FASI) Score | Week 12 FASI Total Score | 5.57 score on a scale | Standard Deviation 0.98 |
| Sirolimus 0.2% | Facial Angifibromas Severity Index (FASI) Score | Open-label week 12 FASI Total Score | 5 score on a scale | Standard Deviation 1 |
| Sirolimus 0.4% | Facial Angifibromas Severity Index (FASI) Score | Open-label week 12 FASI Total Score | 6 score on a scale | Standard Deviation 0.63 |
| Sirolimus 0.4% | Facial Angifibromas Severity Index (FASI) Score | Baseline FASI Total Score | 7.38 score on a scale | Standard Deviation 1.19 |
| Sirolimus 0.4% | Facial Angifibromas Severity Index (FASI) Score | Week 12 FASI Total Score | 5.75 score on a scale | Standard Deviation 1.39 |
| Sirolimus 0.4% | Facial Angifibromas Severity Index (FASI) Score | Week 4 FASI Total Score | 6.5 score on a scale | Standard Deviation 1.69 |
| Placebo | Facial Angifibromas Severity Index (FASI) Score | Open-label week 12 FASI Total Score | 5.33 score on a scale | Standard Deviation 1.51 |
| Placebo | Facial Angifibromas Severity Index (FASI) Score | Week 4 FASI Total Score | 7.33 score on a scale | Standard Deviation 1.32 |
| Placebo | Facial Angifibromas Severity Index (FASI) Score | Week 12 FASI Total Score | 7 score on a scale | Standard Deviation 1.58 |
| Placebo | Facial Angifibromas Severity Index (FASI) Score | Baseline FASI Total Score | 7.89 score on a scale | Standard Deviation 1.27 |
Secondary
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
Population: \[Not Specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | 0 = No change from baseline | 2 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -2 = 2-point improvement from baseline | 1 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | 0 = No change from baseline | 4 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.2% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -1 = 1-point improvement from baseline | 3 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | 0 = No change from baseline | 4 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | 0 = No change from baseline | 3 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -1 = 1-point improvement from baseline | 4 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | 0 = No change from baseline | 4 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -1 = 1-point improvement from baseline | 2 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -1 = 1-point improvement from baseline | 4 Participants |
| Sirolimus 0.4% | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -2 = 2-point improvement from baseline | 1 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | 0 = No change from baseline | 8 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -1 = 1-point improvement from baseline | 1 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 4 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | 0 = No change from baseline | 9 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -2 = 2-point improvement from baseline | 0 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | 0 = No change from baseline | 3 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from baseline in lesion elevation: Open-label week 12 | -1 = 1-point improvement from baseline | 3 Participants |
| Placebo | The Change From Baseline in Lesion Elevation | Change from Baseline in lesion elevation: Week 12 | -1 = 1-point improvement from baseline | 0 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | Yes (N) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | Yes (N) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | No (N) | 4 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | Yes (N) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | Yes (N) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | No (N) | 7 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | Yes (N) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | Yes (N) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | No (N) | 7 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | Yes (N) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | Yes (N) | 2 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 12 | Yes (N) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Week 4 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | >=2 grade improvement in lesion: Open-label week 12 | No (N) | 4 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | No (N) | 4 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | Yes (Y) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | Yes (Y) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | Yes (Y) | 0 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | Yes (Y) | 0 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | No (N) | 8 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | No (N) | 8 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 4 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Week 12 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | >=2 grade improvement in lesion elevation: Open-label week 12 | No (N) | 6 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | No (N) | 6 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | Yes (Y) | 1 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | No (N) | 5 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | Yes (Y) | 2 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | No (N) | 5 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | Yes (Y) | 2 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | No (N) | 3 Participants |
| Sirolimus 0.2% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | Yes (Y) | 3 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | No (N) | 6 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | No (N) | 3 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | Yes (Y) | 2 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | No (N) | 2 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | Yes (Y) | 4 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | No (N) | 7 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | Yes (Y) | 3 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | No (N) | 8 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 4 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: Week 12 | Yes (Y) | 1 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | No (N) | 4 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | Yes (Y) | 1 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 4 | No (N) | 8 Participants |
| Placebo | The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | >=2 grade improvement in self-assessment: open-label week 12 | Yes (Y) | 3 Participants |
Secondary
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
Population: \[Not specified\]
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | No (N) | 3 Participants |
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | Yes (Y) | 2 Participants |
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | No (N) | 5 Participants |
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | Yes (Y) | 2 Participants |
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | No (N) | 5 Participants |
| Sirolimus 0.2% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | Yes (Y) | 2 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | No (N) | 5 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | Yes (Y) | 1 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | No (N) | 6 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | Yes (Y) | 2 Participants |
| Sirolimus 0.4% | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | Yes (Y) | 3 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | Yes (Y) | 0 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | No (N) | 3 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 12 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >=30% Improvement in FASI Score Week 4 | No (N) | 9 Participants |
| Placebo | The Percentage of Subjects With at Least 30% Improvement in the Facial Angiofibromas Severity Index (FASI) Score. | >= 30% Improvement in FASI Score: Open-label week 12 | Yes (Y) | 3 Participants |