Trial · NCT03363763
Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex
NCT03363763, Phase 2, Terminated, Aucta Pharmaceuticals, Inc; results first posted 2025-12-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex. Trials, record NCT03363763. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03363763
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex
- NCT number
- NCT03363763
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Aucta Pharmaceuticals, Inc
- Conditions
- Angiofibroma of Face, Tuberous Sclerosis
- Interventions
- Sirolimus 0.2%, Sirolimus 0.4%, Placebo ointment
- First posted
- 2017-12-06
- Last updated
- 2025-12-01
- Results first posted
- 2025-12-01
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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. |
| Primary | 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 | 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. |
| Secondary | 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. |
| Secondary | 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 \[0 = no lesion, 1 = \<25 lesions, 2 = 25 to 50 lesions, 3 = 51 to 75 lesions, 4 = \> 75 lesions\] |
| Secondary | 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) |
| Secondary | 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). |
| Secondary | 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. |
| Secondary | 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) |
| Secondary | 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) |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Percentage of Participants Achieved at Least 2-grade Improvement | Double-blind phase and Open-label phase Weeks 4 and 12 | 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). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | The Change in Baseline in Investigator's Global Assessment (IGA) by Visit | Double-blind phase and Open-label phase Weeks 4 and 12 | 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). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | 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 | \[Not specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Facial Angifibromas Severity Index (FASI) Score | Baseline, Double blind phase weeks 4 and 12 and Open-label week 12 | \[Not specified\] | score on a scale | MEAN |
| SECONDARY | 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 | \[Not specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Lesion Counts | Double blind phase weeks 4 and 12 and open-label phase week 12 | \[Not Specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | 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 | \[Not specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Change From Baseline in Lesion Elevation | Double blind phase weeks 4 and 12 and open-label phase week 12 | \[Not Specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | 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 | \[Not specified\] | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Self-Assessment | Double blind phase weeks 4 and 12 and open label phase weeks 4 and 12 | \[Not specified\] | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group285
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.2% | Primary Endpoint (>=2 point improvement) Week 12 / No (N) | 6 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.4% | Primary Endpoint (>=2 point improvement) Week 12 / No (N) | 6 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Placebo | Primary Endpoint (>=2 point improvement) Week 12 / No (N) | 9 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.2% | Primary Endpoint (>=2 point improvement) Week 12 / Yes (Y) | 1 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.4% | Primary Endpoint (>=2 point improvement) Week 12 / Yes (Y) | 2 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Placebo | Primary Endpoint (>=2 point improvement) Week 12 / Yes (Y) | 0 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.2% | Primary Endpoint (>=2 point improvement) Week 4 / No (N) | 6 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.4% | Primary Endpoint (>=2 point improvement) Week 4 / No (N) | 8 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Placebo | Primary Endpoint (>=2 point improvement) Week 4 / No (N) | 9 | Participants | |
| The Percentage of Participants Achieved at Least 2-grade Improvement | Sirolimus 0.2% | Primary Endpoint (>=2 point improvement) Week 4 / Yes (Y) | 1 | Participants |
1 to 10 of 285Source ClinicalTrials.gov
See all 285 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-Blind Phase | STARTED | Sirolimus 0.2% | 7 | |
| Double-Blind Phase | STARTED | Sirolimus 0.4% | 8 | |
| Double-Blind Phase | STARTED | Placebo | 9 | |
| Double-Blind Phase | COMPLETED | Sirolimus 0.2% | 7 | |
| Double-Blind Phase | COMPLETED | Sirolimus 0.4% | 8 | |
| Double-Blind Phase | COMPLETED | Placebo | 9 | |
| Double-Blind Phase | NOT COMPLETED | Sirolimus 0.2% | 0 | |
| Double-Blind Phase | NOT COMPLETED | Sirolimus 0.4% | 0 | |
| Double-Blind Phase | NOT COMPLETED | Placebo | 0 | |
| Open-label Phase | STARTED | Sirolimus 0.2% | 7 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events186
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Double-Blind: Sirolimus 0.2% | 0 | 7 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Double-Blind: Sirolimus 0.4% | 0 | 8 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Double-Blind: Placebo | 1 | 9 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Open-label: Sirolimus 0.2% | 0 | 7 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Open-label: Sirolimus 0.4% | 0 | 7 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Open-label: Placebo | 0 | 9 | 0 |
| other | Mouth Ulceration | Gastrointestinal disorders | Double-Blind: Sirolimus 0.2% | 1 | 7 | 1 |
| other | Mouth Ulceration | Gastrointestinal disorders | Double-Blind: Sirolimus 0.4% | 0 | 8 | 0 |
| other | Mouth Ulceration | Gastrointestinal disorders | Double-Blind: Placebo | 0 | 9 | 0 |
| other | Mouth Ulceration | Gastrointestinal disorders | Open-label: Sirolimus 0.2% | 0 | 7 | 0 |
1 to 10 of 186Source ClinicalTrials.gov
See all 186 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Double-Blind: Sirolimus 0.2% | 0 | 7 |
| Total, all-cause mortality | Double-Blind: Sirolimus 0.4% | 0 | 8 |
| Total, all-cause mortality | Double-Blind: Placebo | 0 | 9 |
| Total, all-cause mortality | Open-label: Sirolimus 0.2% | 0 | 7 |
| Total, all-cause mortality | Open-label: Sirolimus 0.4% | 0 | 7 |
| Total, all-cause mortality | Open-label: Placebo | 0 | 9 |
| Total, other adverse events | Double-Blind: Sirolimus 0.2% | 6 | 7 |
| Total, other adverse events | Double-Blind: Sirolimus 0.4% | 5 | 8 |
| Total, other adverse events | Double-Blind: Placebo | 4 | 9 |
| Total, other adverse events | Open-label: Sirolimus 0.2% | 1 | 7 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03363763
GET https://conductscience.org/api/trials/v1/NCT03363763/results?page=2