Trial · NCT02152007
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
NCT02152007, Phase 1, Completed, TransDerm, Inc.; results first posted 2016-10-07
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC). Trials, record NCT02152007. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02152007
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
- NCT number
- NCT02152007
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 15 participants, actual
- Lead sponsor
- TransDerm, Inc.
- Conditions
- Pachyonychia Congenita
- Interventions
- 1% sirolimus cream (TD201 1%)
- First posted
- 2014-06-02
- Last updated
- 2016-10-07
- Results first posted
- 2016-10-07
- Limitations and caveats
- This study was primarily concerned with safety endpoints. Efficacy endpoints need to be improved for follow on studies.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug | The primary outcome measure for this Phase 1b safety study is evaluation of system absorption through measurement of serum sirolimus trough levels. The limit of detection of the assay was 2.0 ng/mL. |
| Secondary | Weekly Assessments Recorded in the PC Quality of Life Index | Weekly for 39 weeks | Patient-reported weekly assessment in the PC Quality of Life Index |
| Secondary | Daily Assessments Recording in the PC Measurement Diary | Weekly for 39 weeks | |
| Other | Investigator Assessment of Local Tolerability | Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks | Investigator assessment of local tolerability at the application sites on the plantar surfaces will be evaluated by the Investigator according to a 4-point scale (0, 1, 2, or 3; none to severe) with regard to: erythema, pruritis, stinging/burning, and crusting/erosion |
| Other | Standardized Photographs | Each study visit over 39 weeks | An expert in the disease who is blinded to the study treatment will read the photographs of the callus area taken at each study visit. The reader will assess changes to the calluses based on criteria such as blisters, cracks, small/large size, and red or bloody spots on the callus. Change in calluses will be reported for both the right and left foot. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug | Starting at week 13, visit 4, there were only 14 participants with available data. | participants | NUMBER |
| SECONDARY | Weekly Assessments Recorded in the PC Quality of Life Index | Weekly for 39 weeks | |||
| SECONDARY | Daily Assessments Recording in the PC Measurement Diary | Weekly for 39 weeks | |||
| OTHER_PRE_SPECIFIED | Investigator Assessment of Local Tolerability | Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks | units on a scale | MEAN | |
| OTHER_PRE_SPECIFIED | Standardized Photographs | Each study visit over 39 weeks |
Source ClinicalTrials.gov
Results by group47
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 1, Baseline, <2.0 ng/mL | 15 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 2, Week 3, <2 ng/mL | 15 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 3, Week 7, <2 ng/mL | 15 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 4, Week 13, <2 ng/mL | 14 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 5, Week 17, <2 ng/mL | 14 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 6, Week 25, <2 ng/mL | 14 | participants | |
| Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 7, Week 37, <2 ng/mL | 14 | participants | |
| Investigator Assessment of Local Tolerability | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 1 Pre, Erythema Score (0-3) | 0.2 | Standard Deviation 0.41 | units on a scale |
| Investigator Assessment of Local Tolerability | Placebo Cream (Vehicle Control) | Visit 1 Pre, Erythema Score (0-3) | 0.2 | Standard Deviation 0.41 | units on a scale |
| Investigator Assessment of Local Tolerability | Split-body 1% Sirolimus Cream (TD201 1%) | Visit 1 Pre, Pruritus Score (0-3) | 0.1 | Standard Deviation 0.26 | units on a scale |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Split-body 1% Sirolimus Cream (TD201 1%) | 15 | |
| Overall Study | COMPLETED | Split-body 1% Sirolimus Cream (TD201 1%) | 14 | |
| Overall Study | NOT COMPLETED | Split-body 1% Sirolimus Cream (TD201 1%) | 1 |
Source ClinicalTrials.gov
Adverse events9
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 | Nausea | Gastrointestinal disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Pyrexia | General disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Upper respiratory tract infection | Infections and infestations | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Musculoskeletal pain | Musculoskeletal and connective tissue disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Dermatitis contact | Skin and subcutaneous tissue disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Skin ulcer | Skin and subcutaneous tissue disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Urticaria | Skin and subcutaneous tissue disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Headache | Nervous system disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
| other | Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Split-body 1% Sirolimus Cream (TD201 1%) | 1 | 15 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Split-body 1% Sirolimus Cream (TD201 1%) | ||
| Total, other adverse events | Split-body 1% Sirolimus Cream (TD201 1%) | 6 | 15 |
| Total, serious adverse events | Split-body 1% Sirolimus Cream (TD201 1%) | 0 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT02152007
GET https://conductscience.org/api/trials/v1/NCT02152007/results?page=2