Trial · NCT04154293
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
NCT04154293, Phase 2, Completed, LEO Pharma; results first posted 2022-08-25
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis. Trials, record NCT04154293. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04154293
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
- NCT number
- NCT04154293
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- LEO Pharma
- Conditions
- Congenital Ichthyosis
- Interventions
- Isotretinoin, Vehicle
- First posted
- 2019-11-06
- Last updated
- 2026-05-22
- Results first posted
- 2022-08-25
- Limitations and caveats
- small sample size. study not powered for statistical significance.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement | 12 weeks | Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score |
| Secondary | Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment | 12 weeks | Number of subjects with Investigator Global Assessment decrease in disease severity by at least 2 grades |
| Secondary | Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 | 12 weeks | Mean numeric Change from baseline in Itch-Numeric Rating Scale (I-NRS) at Week 12 for entire population. I-NRS is an 11-point visual analogue scale scored based upon 0 being no itch and 10 being worst itch imaginable. Subjects were asked to grade their itch on such a scale at each visit where I-NRS was administered. A 4-point change from baseline is considered clinically significant |
| Secondary | Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12 | 12 weeks | Change from Baseline in Dermatology Life Quality Index (DLQI) at Week 12 in all adult subjects. The DLQI is validated in all subjects over age 16 years and is a 10-question questionnaire which requests information on how the dermatological condition affects different aspects of their life. Each question is scored on a 0-3 scale (0=no impact, 3=major impact) for a total possible maximal score of 30. Scores over 11 in adults are considered to indicate moderate impairment of quality of life due to the skin disease. A 4-point improvement from baseline is considered clinically significant |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement | 12 weeks | Intention to treat analysis only on subjects who received at least one dose of study medication; one subject in TMB-001 0.1% arm was randomized but never received study medication due to COVID | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment | 12 weeks | Intention to treat analysis only on subjects who received at least one dose of study medication; one subject in TMB-001 0.1% arm was randomized but never received study medication due to COVID | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 | 12 weeks | Intention to treat analysis based upon available data collected. Data imputation was not performed for I-NRS but only for primary endpoint as above | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12 | 12 weeks | Intention to treat analysis based upon available data collected. Data imputation was not performed for DLQI but only for primary endpoint as above | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement | Vehicle Ointment (Control) | 4 | Participants | ||
| Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement | TMB-001 Ointment, 0.05% | 7 | Participants | ||
| Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement | TMB-001 Ointment, 0.1% | 4 | Participants | ||
| Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment | Vehicle Ointment (Control) | 1 | Participants | ||
| Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment | TMB-001 Ointment, 0.05% | 6 | Participants | ||
| Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment | TMB-001 Ointment, 0.1% | 4 | Participants | ||
| Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 | Vehicle Ointment (Control) | -1.7 | Standard Deviation 2.54 | units on a scale | |
| Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 | TMB-001 Ointment, 0.05% | -1.9 | Standard Deviation 5.3 | units on a scale | |
| Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 | TMB-001 Ointment, 0.1% | 1.1 | Standard Deviation 2.64 | units on a scale | |
| Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12 | Vehicle Ointment (Control) | -3.4 | Standard Deviation 4.72 | units on a scale |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Vehicle Ointment (Control) | 12 | |
| Overall Study | STARTED | TMB-001 Ointment, 0.05% | 11 | |
| Overall Study | STARTED | TMB-001 Ointment, 0.1% | 11 | |
| Overall Study | Number Who Received at Least 1 Dose | Vehicle Ointment (Control) | 12 | |
| Overall Study | Number Who Received at Least 1 Dose | TMB-001 Ointment, 0.05% | 11 | |
| Overall Study | Number Who Received at Least 1 Dose | TMB-001 Ointment, 0.1% | 10 | |
| Overall Study | COMPLETED | Vehicle Ointment (Control) | 10 | |
| Overall Study | COMPLETED | TMB-001 Ointment, 0.05% | 8 | |
| Overall Study | COMPLETED | TMB-001 Ointment, 0.1% | 9 | |
| Overall Study | NOT COMPLETED | Vehicle Ointment (Control) | 2 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events66
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 | palipations | Cardiac disorders | Vehicle Ointment (Control) | 0 | 12 | 0 |
| other | palipations | Cardiac disorders | TMB-001 Ointment, 0.05% | 1 | 11 | 1 |
| other | palipations | Cardiac disorders | TMB-001 Ointment, 0.1% | 0 | 10 | 0 |
| other | ear pain | Ear and labyrinth disorders | Vehicle Ointment (Control) | 0 | 12 | 0 |
| other | ear pain | Ear and labyrinth disorders | TMB-001 Ointment, 0.05% | 0 | 11 | 0 |
| other | ear pain | Ear and labyrinth disorders | TMB-001 Ointment, 0.1% | 1 | 10 | 1 |
| other | application site dermatitis | General disorders | Vehicle Ointment (Control) | 0 | 12 | 0 |
| other | application site dermatitis | General disorders | TMB-001 Ointment, 0.05% | 3 | 11 | 6 |
| other | application site dermatitis | General disorders | TMB-001 Ointment, 0.1% | 1 | 10 | 2 |
| other | application site discomfort | General disorders | Vehicle Ointment (Control) | 1 | 12 | 1 |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Vehicle Ointment (Control) | 0 | 12 |
| Total, all-cause mortality | TMB-001 Ointment, 0.05% | 0 | 11 |
| Total, all-cause mortality | TMB-001 Ointment, 0.1% | 0 | 10 |
| Total, other adverse events | Vehicle Ointment (Control) | 3 | 12 |
| Total, other adverse events | TMB-001 Ointment, 0.05% | 9 | 11 |
| Total, other adverse events | TMB-001 Ointment, 0.1% | 8 | 10 |
| Total, serious adverse events | Vehicle Ointment (Control) | 0 | 12 |
| Total, serious adverse events | TMB-001 Ointment, 0.05% | 0 | 11 |
| Total, serious adverse events | TMB-001 Ointment, 0.1% | 0 | 10 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37170057 | DERIVED |
Source PubMed
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD006079 | Ph2.Study of PAT-001 for Treatment of Congenital Ichthyosis IND122058(7/27/2016) | ROME, ZACHARY | TIMBER PHARMACEUTICALS, LLC | FOOD AND DRUG ADMINISTRATION | 2018 to 2021 |
Source NIH RePORTER
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/NCT04154293
GET https://conductscience.org/api/trials/v1/NCT04154293/events?page=2