Trial · NCT02960997

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

NCT02960997, Phase 2, Completed, Stanford University; results first posted 2020-01-30

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study. Trials, record NCT02960997. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02960997

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
NCT number: NCT02960997
Status: Completed
Phases: Phase 2
Enrollment: 8 participants, actual
Lead sponsor: Stanford University
Conditions: Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome
Interventions: Sirolimus, 2%, Vehicle
First posted: 2016-11-10
Last updated: 2022-03-09
Results first posted: 2020-01-30

Source ClinicalTrials.gov

Registered outcomes10

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Foot Health Status Questionnaire, Foot Function Domain Score	Week 0 and week 12 of the respective treatment period	Foot function was assessed utilizing the validated Foot Health Status Questionnaire (FHSQ). Low Score (0) means severely limited in performing a broad range of physical activities. High Score (100) means can perform all desired physical activities. Data are reported per intervention.
Primary	Foot Health Status Questionnaire, Physical Activity Domain Score	Week 0 and week 12 of the respective treatment period	Physical Activity was assessed utilizing the validated Foot Health Status Questionnaire (FHSQ). Low Score (0) means severely limited in performing a broad range of physical activities. High Score (100) means can perform all desired physical activities. Data are reported per intervention.
Primary	Trough Concentration of Sirolimus	Week 12	Trough measurements were taken prior to topical sirolimus administration at the week 12 study visit.
Secondary	Average Steps Per Day Assessed by FitBit® / Pedometer	12 weeks	Average number of steps walked per day from baseline to the end of each treatment.
Secondary	Child Dermatological Quality of Life Questionnaire Score	Week 0 and week 12 of the respective treatment period	Quality of Life-Epidermolysis Bullosa (QOLEB) Questionnaire specifically designed for people with EB. The QOLEB can be used to identify everyday life occurrences negatively affected by EB. It assesses change in quality of life over time, an important measure when assessing the success of new treatments for EB. Scores from 0 to 51, with higher scores indicate greater impact of EB on quality of life. Data are reported per intervention.
Secondary	Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) Disease Severity Scale Score	Week 0 and week 12 of the respective treatment period	The EBDASI is a validated scoring system that objectively quantifies the severity of EB affecting the entire body. It has been designed to evaluate the response to new therapies for the treatment of EB. Scores range from 0 (absent of EB) to 10 (entire area involved). Data are reported per intervention.
Secondary	5-D Pruritus Scale Score	Week 0 and week 12 of the respective treatment period	The 5-D Pruritus Scale is a 1-page, 5-question tool used in clinical trials to assess 5 dimensions of background itch: degree, duration, direction, disability, and distribution. Each question corresponds to 1 of the 5 dimensions of itch; participants were to rate their symptoms over the preceding 2-week period on a 1 to 5 scale, with 5 being the most affected. After the summation of individual score, the total score ranges from 5 (least affected) to 25 (most affected). Data are reported per intervention.
Secondary	Plantar Defect Size Using 3D Photography	Baseline, week 12	Plantar defect size measurements using 3D photography (% change in total defect area).
Secondary	Foot Plantar Pressure Measurements	Baseline, week 12	Foot plantar pressure measurements before and after treatment using the Podotech Elftman Foot Scanner.
Secondary	Change in mTOR Pathway Inhibition	Baseline, week 12	Molecular biology study of skin biopsies assayed for mTOR pathway inhibition (e.g. determination of phosphoprotein inhibition included ribosomal protein S6, S6 kinase and/or eIF-4E binding protein).

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	Foot Health Status Questionnaire, Foot Function Domain Score	Week 0 and week 12 of the respective treatment period		score on a scale	MEAN
PRIMARY	Foot Health Status Questionnaire, Physical Activity Domain Score	Week 0 and week 12 of the respective treatment period		score on a scale	MEAN
PRIMARY	Trough Concentration of Sirolimus	Week 12	Trough concentration of sirolimus was collected during Sirolimus treatment only, so the Placebo group is not presented in this outcome measure.	ng/mL	MEAN
SECONDARY	Average Steps Per Day Assessed by FitBit® / Pedometer	12 weeks	One participant had no Fitbit data during placebo treatment and is excluded from the analysis. Three participants lost their Fitbit devices for week 12 reporting during sirolimus treatment; their data through week 8 are included in the analysis.	steps	MEAN
SECONDARY	Child Dermatological Quality of Life Questionnaire Score	Week 0 and week 12 of the respective treatment period		score on a scale	MEAN
SECONDARY	Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) Disease Severity Scale Score	Week 0 and week 12 of the respective treatment period		score on a scale	MEAN
SECONDARY	5-D Pruritus Scale Score	Week 0 and week 12 of the respective treatment period		score on a scale	MEAN
SECONDARY	Plantar Defect Size Using 3D Photography	Baseline, week 12	Data were not collected for this outcome measure.
SECONDARY	Foot Plantar Pressure Measurements	Baseline, week 12	Data were not collected for this outcome measure.
SECONDARY	Change in mTOR Pathway Inhibition	Baseline, week 12	Data were not collected for this outcome measure

Source ClinicalTrials.gov

Results by group23

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Foot Health Status Questionnaire, Foot Function Domain Score	Sirolimus	Week 0	61.7	Standard Deviation 26.48	score on a scale
Foot Health Status Questionnaire, Foot Function Domain Score	Placebo	Week 0	49.3	Standard Deviation 20.83	score on a scale
Foot Health Status Questionnaire, Foot Function Domain Score	Sirolimus	Week 12	60.2	Standard Deviation 23.37	score on a scale
Foot Health Status Questionnaire, Foot Function Domain Score	Placebo	Week 12	56.3	Standard Deviation 24.09	score on a scale
Foot Health Status Questionnaire, Physical Activity Domain Score	Sirolimus	Week 0	69.4	Standard Deviation 24.37	score on a scale
Foot Health Status Questionnaire, Physical Activity Domain Score	Placebo	Week 0	54.2	Standard Deviation 21.65	score on a scale
Foot Health Status Questionnaire, Physical Activity Domain Score	Sirolimus	Week 12	70.8	Standard Deviation 20.37	score on a scale
Foot Health Status Questionnaire, Physical Activity Domain Score	Placebo	Week 12	61.8	Standard Deviation 24.91	score on a scale
Trough Concentration of Sirolimus	Sirolimus		NA	Standard Deviation NA	ng/mL
Average Steps Per Day Assessed by FitBit® / Pedometer	Sirolimus		6624	Standard Deviation 1847	steps

1 to 10 of 23Source ClinicalTrials.gov

See all 23 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
First Intervention, 12 Weeks	STARTED	Sirolimus, Then Placebo	4
First Intervention, 12 Weeks	STARTED	Placebo, Then Sirolimus	4
First Intervention, 12 Weeks	COMPLETED	Sirolimus, Then Placebo	4
First Intervention, 12 Weeks	COMPLETED	Placebo, Then Sirolimus	4
First Intervention, 12 Weeks	NOT COMPLETED	Sirolimus, Then Placebo	0
First Intervention, 12 Weeks	NOT COMPLETED	Placebo, Then Sirolimus	0
Washout Period, 4 Weeks	STARTED	Sirolimus, Then Placebo	4
Washout Period, 4 Weeks	STARTED	Placebo, Then Sirolimus	4
Washout Period, 4 Weeks	COMPLETED	Sirolimus, Then Placebo	4
Washout Period, 4 Weeks	COMPLETED	Placebo, Then Sirolimus	4

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events38

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
other	Allergic Reaction to antiobiotic (sulfamethoxole)	Immune system disorders	Sirolimus	0	8
other	Allergic Reaction to antiobiotic (sulfamethoxole)	Immune system disorders	Placebo	1	8
other	Burning sensation on feet	Skin and subcutaneous tissue disorders	Sirolimus	1	8
other	Burning sensation on feet	Skin and subcutaneous tissue disorders	Placebo	0	8
other	Common cold	Infections and infestations	Sirolimus	0	8
other	Common cold	Infections and infestations	Placebo	3	8
other	Cough	General disorders	Sirolimus	0	8
other	Cough	General disorders	Placebo	2	8
other	Ear infection	Infections and infestations	Sirolimus	2	8
other	Ear infection	Infections and infestations	Placebo	0	8

1 to 10 of 38Source ClinicalTrials.gov

See all 38 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sirolimus	0	8
Total, all-cause mortality	Placebo	0	8
Total, other adverse events	Sirolimus	8	8
Total, other adverse events	Placebo	8	8
Total, serious adverse events	Sirolimus	0	8
Total, serious adverse events	Placebo	0	8

Source ClinicalTrials.gov

Papers14

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
11416184	BACKGROUND	PubMed
11821896	BACKGROUND	PubMed
11840159	BACKGROUND	PubMed
14987264	BACKGROUND	PubMed
15495218	BACKGROUND	PubMed
19681875	BACKGROUND	PubMed
19699613	BACKGROUND	PubMed
19995367	BACKGROUND	PubMed
22588747	BACKGROUND	PubMed
24075228	BACKGROUND	PubMed

1 to 10 of 14

See all 14 papers

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Sirolimus vs Placebo: deaths from any cause	Trial comparisons
Sirolimus vs Placebo: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT02960997

GET https://conductscience.org/api/trials/v1/NCT02960997/results?page=2

API reference For AI assistants