Trial · NCT01526356
Topical Rapamycin to Erase Angiofibromas in TSC
NCT01526356, Phase 2, Completed, The University of Texas Health Science Center, Houston; results first posted 2020-10-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Topical Rapamycin to Erase Angiofibromas in TSC. Trials, record NCT01526356. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01526356
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Topical Rapamycin to Erase Angiofibromas in TSC
- NCT number
- NCT01526356
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 179 participants, actual
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Conditions
- Angiofibromas, Tuberous Sclerosis
- Interventions
- Placebo, Rapamycin, Rapamycin
- First posted
- 2012-02-03
- Last updated
- 2020-10-26
- Results first posted
- 2020-10-26
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | baseline, 6 months | Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome. |
| Secondary | Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | baseline, 6 months | |
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) | baseline, 6 months | The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life). |
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI) | baseline, 6 months | The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life). |
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI) | baseline, 6 months | The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life). |
| Other | Number of Events of Dermatologic Sensitivity at the Site of Application | 6 months | Dermatologic sensitivity includes pain, pruritis, or erythema at the application site. |
| Other | Number of Participants With Systemic Uptake of Topically Applied Rapamycin | 6 months | Blood levels checked to confirm the lack of systemic rapamycin. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | baseline, 6 months | This data was not collected for 12 in the 1% rapamycin arm, 17 in the 0.1% rapamycin arm, and 17 in the placebo arm. | score on a scale | MEAN |
| SECONDARY | Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | baseline, 6 months | This data was not collected for 4 in the 1% rapamycin arm, 5 in the 0.1% rapamycin arm, and 6 in the placebo arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) | baseline, 6 months | This data was not collected for 29 in the 1% rapamycin arm, 34 in the 0.1% rapamycin arm, and 35 in the placebo arm. | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI) | baseline, 6 months | This data was not collected for 46 in the 1% rapamycin arm, 57 in the 0.1% rapamycin arm, and 48 in the placebo arm. | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI) | baseline, 6 months | This data was not collected for 41 in the 1% rapamycin arm, 35 in the 0.1% rapamycin arm, and 38 in the placebo arm. | score on a scale | MEAN |
| OTHER_PRE_SPECIFIED | Number of Events of Dermatologic Sensitivity at the Site of Application | 6 months | number of events | NUMBER | |
| OTHER_PRE_SPECIFIED | Number of Participants With Systemic Uptake of Topically Applied Rapamycin | 6 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | 1% Rapamycin | -13.89 | Standard Deviation 20.86 | score on a scale | |
| Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | 0.1 % Rapamycin | -12.71 | Standard Deviation 20.24 | score on a scale | |
| Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | Placebo | -1.39 | Standard Deviation 14.64 | score on a scale | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | 1% Rapamycin | better | 45 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | 0.1 % Rapamycin | better | 38 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | Placebo | better | 13 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | 1% Rapamycin | same | 5 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | 0.1 % Rapamycin | same | 11 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | Placebo | same | 15 | Participants | |
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | 1% Rapamycin | worse | 5 | Participants |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 1% Rapamycin | 59 | |
| Overall Study | STARTED | 0.1 % Rapamycin | 63 | |
| Overall Study | STARTED | Placebo | 57 | |
| Overall Study | COMPLETED | 1% Rapamycin | 51 | |
| Overall Study | COMPLETED | 0.1 % Rapamycin | 53 | |
| Overall Study | COMPLETED | Placebo | 45 | |
| Overall Study | NOT COMPLETED | 1% Rapamycin | 8 | |
| Overall Study | NOT COMPLETED | 0.1 % Rapamycin | 10 | |
| Overall Study | NOT COMPLETED | Placebo | 12 |
Source ClinicalTrials.gov
Adverse events60
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 | Application site pain | Skin and subcutaneous tissue disorders | Placebo | 1 | 59 | 1 |
| other | Application site pain | Skin and subcutaneous tissue disorders | 0.1 % Rapamycin | 2 | 63 | 2 |
| other | Application site pain | Skin and subcutaneous tissue disorders | 1% Rapamycin | 6 | 57 | 6 |
| other | Application site pruritus | Skin and subcutaneous tissue disorders | Placebo | 1 | 59 | 1 |
| other | Application site pruritus | Skin and subcutaneous tissue disorders | 0.1 % Rapamycin | 0 | 63 | 0 |
| other | Application site pruritus | Skin and subcutaneous tissue disorders | 1% Rapamycin | 5 | 57 | 5 |
| other | Application site acne | Skin and subcutaneous tissue disorders | Placebo | 1 | 59 | 1 |
| other | Application site acne | Skin and subcutaneous tissue disorders | 0.1 % Rapamycin | 1 | 63 | 1 |
| other | Application site acne | Skin and subcutaneous tissue disorders | 1% Rapamycin | 3 | 57 | 3 |
| other | Cutaneous eruption | Skin and subcutaneous tissue disorders | Placebo | 1 | 59 | 1 |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 59 |
| Total, all-cause mortality | 0.1 % Rapamycin | 0 | 63 |
| Total, all-cause mortality | 1% Rapamycin | 0 | 57 |
| Total, other adverse events | Placebo | 10 | 59 |
| Total, other adverse events | 0.1 % Rapamycin | 18 | 63 |
| Total, other adverse events | 1% Rapamycin | 22 | 57 |
| Total, serious adverse events | Placebo | 1 | 59 |
| Total, serious adverse events | 0.1 % Rapamycin | 1 | 63 |
| Total, serious adverse events | 1% Rapamycin | 2 | 57 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 29800048 | DERIVED |
Source PubMed
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/NCT01526356
GET https://conductscience.org/api/trials/v1/NCT01526356/results?page=2