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Terminated (The clinical trial has ended prematurely due to low patient recruitment)
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sirolimus 0.2% | Active Comparator | Sirolimus 0.2% ointment applied topically hs x 12 weeks |
|
| Sirolimus 0.4% | Active Comparator | Sirolimus 0.4% ointment applied topically hs x 12 weeks |
|
| Placebo | Placebo Comparator | Placebo ointment applied topically hs x 12 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus 0.2% | Drug | Ointment for topical administration hs x 12 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Double-blind phase and Open-label phase Weeks 4 and 12 |
| The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | The Investigator's Global Assessment (IGA) was recorded on a 5-point scale, 0 (minimum) to 5 (maximum) [0 = Clear skin with no signs of erythema and no disease related lesions, 1 = Slight redness with few disease related lesions, 2 = Greater than Grade 1; definite redness with scattered, some disease related lesions, 3 = Greater than Grade 2; marked redness, concentrated, many disease related lesions, 4 = Greater than Grade 3; very bright redness, confluent, highly concentrated disease related lesions, 5= Greater than Grade 4; fiery redness, very extensive disease related lesions covering very large area of the face]. A higher score indicates a more severe, worse outcome. Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement) | Double-blind phase and Open-label phase Weeks 4 and 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Generally healthy males or non-pregnant females aged 2 to 21 years, inclusive, at the time of screening.
Diagnosis of TSC with visible facial angiofibromas of at least grade 3 up to grade 5, inclusive, based on the IGA.
Subjects with 3 or more isolated, measurable lesions of facial angiofibroma, with color grading score ≥2 for each of the 3 lesions.
Females of childbearing potential must have a negative urine pregnancy test (or a negative serum pregnancy test if a urine pregnancy test cannot be obtained) (For China, different pregnancy test would be followed) and if sexually active or become sexually active during the study, must agree to use an effective form of birth control for the duration of the study. Females using oral contraceptives must also use a barrier method of contraception during the study. Sexually active male subjects and/or their female partners should also use appropriate contraception.
Effective contraception is defined as follows:
The subject and/or their parent or guardian must be willing and able to provide written informed consent/assent.
Willing and able to comply with all trial requirements.
Subject or parent/guardian must be able to complete the subject self-assessment survey and subject diary in English or another language into which the documents have been officially translated.
Subjects should be in good general health based on the subject's medical history, physical exam, and impression of the study doctor.
Exclusion Criteria:
Has any chronic or acute medical condition, that in the opinion of the investigator, may pose a risk to the safety of the subject during the trial period, or may interfere with the assessment of safety or efficacy in this trial.
Has received oral therapy or topical therapy of an mTOR inhibitor (sirolimus, temsirolimus, or everolimus) within 1 month of Baseline or other dermatological treatment to facial angiofibromas within 1 month of baseline. (Sunscreen is expected to be used in this patient population and is not considered treatment.)
Is currently receiving any form of immunosuppression therapy or has previously experienced significant immune dysfunction.
Has a history of sensitivity to any component of the investigational product.
Is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
Has other dermatologic conditions, pigmentation, scarring, pigmented lesions or sunburn in the treatment area that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
Has facial hair (e.g., beard, sideburns, mustache) that could interfere with study assessments.
Has had laser surgery or cryotherapy to facial angiofibromas within 6 months preceding study entry.
Requires the use of any concomitant medication that, in the investigator's opinion, has the potential to cause an adverse effect when given with the investigational product or will interfere with the interpretation of the study results (see Section 16.1 Appendix 1 for Potential Drug Interactions).
Has participated in another clinical trial or received an investigational product within 3 months prior to screening.
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| Name | Affiliation | Role |
|---|---|---|
| Shoufeng Li, Ph.D | Aucta Pharmaceuticals, Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Translational Genomics Research | Phoenix | Arizona | 85004 | United States | ||
| Children's Hospital of Los Angeles, Division of Neurology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22934754 | Background | 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. | |
| 23680945 |
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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.
Study subjects were enrolled at 3-5 investigational sites in the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Sirolimus 0.2% | Sirolimus 0.2% ointment applied topically once daily at bedtime for 12 weeks |
| FG001 | Sirolimus 0.4% | Sirolimus 0.4% ointment applied topically once daily at bedtime for 12 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-Blind Phase |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 16, 2021 |
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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.
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| Sirolimus 0.4% | Drug | Ointment for topical administration hs x 12 weeks |
|
|
| Placebo ointment | Drug | Placebo ointment comparator for topical administration hs x 12 weeks |
|
|
| Double-blind phase Weeks 4 and 12 and Open-label phase Week 12 |
| Facial Angifibromas Severity Index (FASI) Score | The Facial Angiofibromas Severity Index (FASI) is derived from the measurements of erythema, average size, and lesion extension. The FASI score is the sum of scores of Erythema, Size, and Extension. Erythema was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = clear skin with no signs of erythema, 1 = almost clear; slight redness, 3= moderate erythema; marked redness, 4= severe erythema; very bright redness]. Size was recorded on a 4-point grade scale from 0 (minimum) to 4 (maximum) [0 = no lesions, 1= few lesions, average size </= 2mm, 2 = scattered, some lesions, average lesion size >2-to </= 5 mm, 3 = concentrated, many lesions, average lesion size > 5mmg to </= 10mm, 4 = confluent, highly concentrated lesions]. Extension was recorded on a grade scale of 0 (minimum), 2, and 3 (maximum) [0 = no lesions, 2 = </= 50% of the cheek surface, 3 = >50% of the cheek surface]. A higher score for Erythema, Size, and Extension means a more severe, worse outcome. | Baseline, Double blind phase weeks 4 and 12 and Open-label week 12 |
| The Percentage of Subjects Achieved at Least 2-grade Improvement in Categorical Lesion Counts by Visit | Lesion counts are measured based on categories 0 to 4 [0 = no lesion, 1 = <25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = > 75 lesions] | Double blind phase Weeks 4 and 12 and Open-label phase Week 12 |
| Change From Baseline in Lesion Counts | Lesion counts are measured based on categories 0 to 4 (0 = no lesion, 1 = <25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = > 75 lesions) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement) | Double blind phase weeks 4 and 12 and open-label phase week 12 |
| The Percentage of Subjects Achieved at Least 2-grade Improvement in Lesion Elevation. | The degree of lesion elevation is assessed on categories 0 to 4 (0 = no elevation over normal skin, 1 = possible but difficult to ascertain whether there is slight elevation above normal skin, 2 = slight but definite elevation, 3 = moderate elevation, 4 = marked elevation). | Double blind phase weeks 4 and 12 and open-label phase week 12 |
| The Change From Baseline in Lesion Elevation | The degree of lesion elevation is assessed on categories 0 to 4 (0 = no elevation over normal skin, 1 = possible but difficult to ascertain whether there is slight elevation above normal skin, 2 = slight but definite elevation, 3 = moderate elevation, 4 = marked elevation) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement) One participant with baseline elevation grade of 1 improved to grade 0 at week 4. This participant is counted as improvement in lesion elevation. This participant was back to grade 1 at week 12 and open-label week 12. | Double blind phase weeks 4 and 12 and open-label phase week 12 |
| The Percentage of Subjects Achieved at Least 2-grade Improvement in the Subject Self-assessment Survey | Subject self- assessment survey was based on categories 0 to 4 (0 = no redness and no disease related lesions, 1 = very mild redness with few very small bumps, 2 = mild redness with many small and medium sized bumps, 3 = moderate redness with many small and medium sized bumps, 4 = severe redness with numerous small, medium, and large sized bumps) | Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12 |
| Change From Baseline in Self-Assessment | Subject self- assessment survey was based on categories 0 to 4 (0 = no redness and no disease related lesions, 1 = very mild redness with few very small bumps, 2 = mild redness with many small and medium sized bumps, 3 = moderate redness with many small and medium sized bumps, 4 = severe redness with numerous small, medium, and large sized bumps) Negative values indicate improvement (0 = no change, -1 = 1-point improvement, -2 = 2-point improvement) | Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12 |
| 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 |
| 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. |
| 21692771 | Background | 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. |
| 23909960 | Background | 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. |
| Background | Rapamune (sirolimus) complete prescribing information. Wyeth Pharmaceuticals Inc. October 2009 |
| FG002 | Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks |
| COMPLETED |
|
| NOT COMPLETED |
|
| Open-label Phase |
|
[Not Specified]
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| ID | Title | Description |
|---|---|---|
| BG000 | Arm 1 | Sirolimus 0.2% ointment applied topically once daily at bedtime for 12 weeks |
| BG001 | Arm 2 | Sirolimus 0.4% ointment applied topically once daily at bedtime for 12 weeks |
| BG002 | Arm 3 | Placebo ointment applied topically once daily at bedtime for 12 weeks |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Height (cm) | Mean | Standard Deviation | cm |
| |||||||||||||||
| Weight (kg) | Mean | Standard Deviation | kg |
| |||||||||||||||
| BMI (kg/m^2) | Mean | Standard Deviation | kg/m^2 |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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). | Posted | Count of Participants | Participants | Double-blind phase and Open-label phase Weeks 4 and 12 |
|
|
| ||||||||||||||||||||||||||||||||
| 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) | 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). | Posted | Count of Participants | Participants | Double-blind phase and Open-label phase Weeks 4 and 12 |
| ||||||||||||||||||||||||||||||||||
| 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. | [Not specified] | Posted | Count of Participants | Participants | Double-blind phase Weeks 4 and 12 and Open-label phase Week 12 |
| ||||||||||||||||||||||||||||||||||
| 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. | [Not specified] | Posted | Mean | Standard Deviation | score on a scale | Baseline, Double blind phase weeks 4 and 12 and Open-label week 12 |
| |||||||||||||||||||||||||||||||||
| 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] | [Not specified] | Posted | Count of Participants | Participants | Double blind phase Weeks 4 and 12 and Open-label phase Week 12 |
|
| |||||||||||||||||||||||||||||||||
| 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) | [Not Specified] | Posted | Count of Participants | Participants | Double blind phase weeks 4 and 12 and open-label phase week 12 |
|
| |||||||||||||||||||||||||||||||||
| 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). | [Not specified] | Posted | Count of Participants | Participants | Double blind phase weeks 4 and 12 and open-label phase week 12 |
|
| |||||||||||||||||||||||||||||||||
| 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. | [Not Specified] | Posted | Count of Participants | Participants | Double blind phase weeks 4 and 12 and open-label phase week 12 |
| ||||||||||||||||||||||||||||||||||
| 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) | [Not specified] | Posted | Count of Participants | Participants | Double blind phase weeks 4 and 12 and open-label phase weeks 4 and 12 |
|
| |||||||||||||||||||||||||||||||||
| 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) | [Not specified] | Posted | Count of Participants | Participants | Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12 |
|
|
30 weeks
Not provided
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Double-Blind: Sirolimus 0.2% | Sirolimus 0.2% ointment applied topically once daily at bedtime for 12 weeks | 0 | 7 | 0 | 7 | 6 | 7 |
| EG001 | Double-Blind: Sirolimus 0.4% | Sirolimus 0.4% ointment applied topically once daily at bedtime for 12 weeks | 0 | 8 | 0 | 8 | 5 | 8 |
| EG002 | Double-Blind: Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks | 0 | 9 | 1 | 9 | 4 | 9 |
| EG003 | Open-label: Sirolimus 0.2% | Sirolimus 0.2% ointment applied topically once daily at bedtime for 12 weeks | 0 | 7 | 1 | 7 | 1 | 7 |
| EG004 | Open-label: Sirolimus 0.4% | Sirolimus 0.4% ointment applied topically once daily at bedtime for 12 weeks | 0 | 7 | 1 | 7 | 5 | 7 |
| EG005 | Open-label: Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks | 0 | 9 | 0 | 9 | 4 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Agitation | Psychiatric disorders | Non-systematic Assessment | Treatment-emergent Serious Adverse Event |
| |
| Ligament rupture | Injury, poisoning and procedural complications | Non-systematic Assessment | Treatment-emergent Serious Adverse Event |
| |
| Psychotic disorder due to a general medical condition | Psychiatric disorders | Non-systematic Assessment | Treatment-emergent Serious Adverse Event |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Mouth Ulceration | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Application site reaction | General disorders | Non-systematic Assessment |
| ||
| Pain | General disorders | Non-systematic Assessment |
| ||
| Pyrexia | General disorders | Non-systematic Assessment |
| ||
| Ear infection | Infections and infestations | Non-systematic Assessment |
| ||
| Respiratory syncytial virus infection | Infections and infestations | Non-systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Non-systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Non-systematic Assessment |
| ||
| Lip injury | Injury, poisoning and procedural complications | Non-systematic Assessment |
| ||
| Epilepsy | Nervous system disorders | Non-systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Non-systematic Assessment |
| ||
| Obsessive thoughts | Psychiatric disorders | Non-systematic Assessment |
| ||
| Sleep disorder | Psychiatric disorders | Non-systematic Assessment |
| ||
| Dsymenorrhoea | Reproductive system and breast disorders | Non-systematic Assessment |
| ||
| Acne | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Skin irritation | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Influenza | Infections and infestations | Non-systematic Assessment |
| ||
| Pharyngitis streptococcal | Infections and infestations | Non-systematic Assessment |
| ||
| Sinusitis | Infections and infestations | Non-systematic Assessment |
| ||
| Renal neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Non-systematic Assessment |
| ||
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| Dermatitis | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Joint injury | Injury, poisoning and procedural complications | Non-systematic Assessment |
| ||
| Tooth Infection | Infections and infestations | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Marie Tan | Aucta Pharmaceuticals, Inc. | 7329129575 | Marie.Tan@auctapharma.com |
| Jun 10, 2025 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D014402 | Tuberous Sclerosis |
| D005076 | Exanthema |
| D005350 | Fibroma |
| D019066 | Facies |
| D004890 | Erythema |
| ID | Term |
|---|---|
| D006222 | Hamartoma |
| D009369 | Neoplasms |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D018218 | Neoplasms, Fibrous Tissue |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Yes (Y) |
|
| Primary Endpoint (>=2 point improvement) Week 4 |
|
|
| Primary Endpoint (>/=2 point improvement) (Open-label Week 4) |
|
|
| Primary Endpoint (>=2 point improvement) (Open-label Week 12) |
|
|
| OG002 | Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks |
|
|
| OG002 | Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks |
|
|
| OG002 | Placebo | Placebo ointment applied topically once daily at bedtime for 12 weeks |
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Participants |
|
|
| Counts |
|---|
| Participants |
|
|
| -1 = 1- point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| -1 = 1- point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| -1 = 1- point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| Yes (Y) |
|
| Yes (Y) |
|
| Yes (N) |
|
| Yes (N) |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| Yes (Y) |
|
| Yes (Y) |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| Yes (Y) |
|
| Yes (Y) |
|
| Yes (Y) |
|
| 0 = No change from baseline |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| 0 = No change from baseline |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|
| 0 = No change from baseline |
|
| -1 = 1-point improvement from baseline |
|
| -2 = 2-point improvement from baseline |
|
| -3 = 3-point improvement from baseline |
|