Congenital IchthyosisPhase 2NCT04154293
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
A Phase 2 study testing Isotretinoin in people with Congenital Ichthyosis, run by LEO Pharma at 11 sites in 2 countries.
Trial summary
The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis… Read more about this study
- Who is it for?
- Ages 9 years and older
- Conditions the study is for
- Congenital Ichthyosis
- What is being tested?
- Isotretinoin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 3 Dec 2019to 30 Aug 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is male or female, 9 years of age or older at Visit 2 (Baseline)
- Subject has provided written informed consent
- Females of child bearing potential must be surgically sterile or agree to 2 forms of birth control
- Subject has clinical diagnosis of Congenital Ichthyosis with genetic confirmation of subtype
- Subject has between 10% and 90% total BSA affected by Congenital Ichthyosis
- Subject has at least 2 VIIS assessment areas with a scaling score of 3 or greater
Who cannot join
- Subject has inflammatory skin diseases unrelated to ichthyosis
- Subject has used other prohibited topical treatments in the assessment areas within certain days from baseline
- Subject has used systemic retinoids within12 weeks of baseline
- Subject has untreated secondary infections
- Subject has lesions suspicious for skin cancer or untreated skin cancers
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States8 sites
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Austin Institute for Clinical Research, Inc
Metairie, Louisiana, 70005
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112
Yale Center for Clinical Investigation
New Haven, Connecticut, 06510
Stanford University School of Medicine
Palo Alto, California, 94304
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19140
Medical Dermatology Specialists
Phoenix, Arizona, 85006
The Indiana Clinical Trials Center
Plainfield, Indiana, 46168
Australia3 sites
Queensland Children's Hospital
Brisbane, Queensland, 4101
Premier Specialists, The church
Kogarah, Sydney, NSW 2217
Murdoch Children's Research Institute, RCH
Melbourne, Victoria, 3052
About this study
Official title: A Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital Ichthyosis
The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.
Funding Source FDA-OOPD
Read the detailed description
This is a randomized, parallel, double-blind, vehicle-controlled study to evaluate the safety and efficacy of two concentrations of topical TMB-001 for the treatment of CI in subjects with either the ARCI-LI or RXLI subtypes. The duration of treatment will be 12 weeks. Each subject will participate in the study for up to 24 weeks (including up to a 90-day Screening period). Eleven study centers from United States of America and Australia participated in this global study. Each subject participated in the study for up to 24 weeks (including up to a 90-day Screening period).
Eligible subjects were randomized (1:1:1) to one of 3 treatment groups:
- TMB-001, 0.05%, twice daily (bid)
- TMB-001, 0.1%, bid
- Vehicle Ointment (Control), 0%, bid
Subjects were evaluated on a monthly basis for safety and efficacy results over the 12- week treatment period. Efficacy was determined in 2 ways:
- VIIS (or Visual Index of Ichthyosis Severity) "treatment success" (VIIS-50) was defined as ≥ 50% reduction from Baseline for VIIS scaling score for the sum of the scores for 'VIIS body areas' that had a Baseline score ≥ 3). The proportion of subjects achieving VIIS-50 at Visit 6 at Week 12 relative to Baseline was the primary efficacy endpoint and time point used to compare the two active treatments to vehicle.
- The proportion of subjects achieving IGA (Investigator Global Assessment) treatment success will be the key secondary endpoint. For this endpoint, the IGA score was established as "treatment success" or "treatment failure" where "treatment success" is defined as at least a 2-grade decrease in severity score (for example, severe or score of 4 at baseline to mild or score of 2 at Visit 6) relative to Baseline at Visit 6 at week 12.
The safety analyses conducted were extent of exposure, changes in physical examinations and vital signs, blood and urine testing including urine pregnancy testing as indicated, local skin reactions (including burning/stinging, redness, erosions, and swelling) and other adverse events.
- Conditions
- Congenital Ichthyosis
- Being tested
- Isotretinoin, Vehicle
- Medical subject headings
- Ichthyosis Vulgaris
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IsotretinoinDrug
Also called TMB-001
Topical Isotretinoin ointment
VehicleOther
Topical Vehicle Ointment
| Study group | Role | What they receive |
|---|---|---|
| Vehicle Ointment (Control) | Placebo comparator | Topical, BID (Twice daily) Receives: Other: Vehicle |
| TMB-001 Ointment, 0.05% | Experimental | Topical, BID ( twice daily) Receives: Drug: Isotretinoin |
| TMB-001 Ointment, 0.1% | Experimental | Topical, BID (Twice daily) Receives: Drug: Isotretinoin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
Measured: 12 weeks
Secondary outcomes (3)
Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment
Number of subjects with Investigator Global Assessment decrease in disease severity by at least 2 grades
Measured: 12 weeks
Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12
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
Measured: 12 weeks
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12
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
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2019
- Study start3 Dec 2019
- Primary completion20 Aug 2021
- Study completion30 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital IchthyosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- A Randomized, Parallel, Double-Blind, Vehicle-Controlled Study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- LEO Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04154293
- Sponsor's protocol ID
- 235-9051-202
- Other IDs
- 1R01FD006079-01A1
- Status last verified
- Aug 2022
- First submitted
- 1 Nov 2019
- Last updated
- 22 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Marathe K, Teng JMC, Guenthner S, Bunick CG, Kempers S, Eads K, Castelo-Soccio L, Mendelsohn AM, Raiz J, Murrell DF. Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study. Dermatol Ther (Heidelb). 2023 Jun;13(6):1255-1264. doi: 10.1007/s13555-023-00923-1. Epub 2023 May 11.
PubMed 37170057
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital Ichthyosis
- ClinicalTrials.gov ID
- NCT04154293
- Acronym / sponsor ID
- 235-9051-202
- Conditions
- Congenital Ichthyosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- LEO PharmaIndustry (pharma, biotech, devices)
- Study start
- 3 Dec 2019 (actual)
- Primary completion
- 20 Aug 2021 (actual)
- Study completion
- 30 Aug 2021 (actual)
- First posted
- 6 Nov 2019 (actual)
- Results
- Posted 25 Aug 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.
Funding Source FDA-OOPD
Detailed description
This is a randomized, parallel, double-blind, vehicle-controlled study to evaluate the safety and efficacy of two concentrations of topical TMB-001 for the treatment of CI in subjects with either the ARCI-LI or RXLI subtypes. The duration of treatment will be 12 weeks. Each subject will participate in the study for up to 24 weeks (including up to a 90-day Screening period). Eleven study centers from United States of America and Australia participated in this global study. Each subject participated in the study for up to 24 weeks (including up to a 90-day Screening period).
Eligible subjects were randomized (1:1:1) to one of 3 treatment groups:
- TMB-001, 0.05%, twice daily (bid)
- TMB-001, 0.1%, bid
- Vehicle Ointment (Control), 0%, bid
Subjects were evaluated on a monthly basis for safety and efficacy results over the 12- week treatment period. Efficacy was determined in 2 ways:
- VIIS (or Visual Index of Ichthyosis Severity) "treatment success" (VIIS-50) was defined as ≥ 50% reduction from Baseline for VIIS scaling score for the sum of the scores for 'VIIS body areas' that had a Baseline score ≥ 3). The proportion of subjects achieving VIIS-50 at Visit 6 at Week 12 relative to Baseline was the primary efficacy endpoint and time point used to compare the two active treatments to vehicle.
- The proportion of subjects achieving IGA (Investigator Global Assessment) treatment success will be the key secondary endpoint. For this endpoint, the IGA score was established as "treatment success" or "treatment failure" where "treatment success" is defined as at least a 2-grade decrease in severity score (for example, severe or score of 4 at baseline to mild or score of 2 at Visit 6) relative to Baseline at Visit 6 at week 12.
The safety analyses conducted were extent of exposure, changes in physical examinations and vital signs, blood and urine testing including urine pregnancy testing as indicated, local skin reactions (including burning/stinging, redness, erosions, and swelling) and other adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- A Randomized, Parallel, Double-Blind, Vehicle-Controlled Study
| Arm | Type | Receives |
|---|---|---|
| Vehicle Ointment (Control) | Placebo comparator | Other: VehicleTopical, BID (Twice daily) |
| TMB-001 Ointment, 0.05% | Experimental | Drug: IsotretinoinTopical, BID ( twice daily) |
| TMB-001 Ointment, 0.1% | Experimental | Drug: IsotretinoinTopical, BID (Twice daily) |
Who may qualify
- Ages
- 9 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Subject is male or female, 9 years of age or older at Visit 2 (Baseline)
- Subject has provided written informed consent
- Females of child bearing potential must be surgically sterile or agree to 2 forms of birth control
- Subject has clinical diagnosis of Congenital Ichthyosis with genetic confirmation of subtype
- Subject has between 10% and 90% total BSA affected by Congenital Ichthyosis
Key exclusion criteria (5 listed)
- Subject has inflammatory skin diseases unrelated to ichthyosis
- Subject has used other prohibited topical treatments in the assessment areas within certain days from baseline
- Subject has used systemic retinoids within12 weeks of baseline
- Subject has untreated secondary infections
- Subject has lesions suspicious for skin cancer or untreated skin cancers
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IsotretinoinAlso: TMB-001Topical Isotretinoin ointment | Drug | TMB-001 Ointment, 0.05%, TMB-001 Ointment, 0.1% |
| VehicleTopical Vehicle Ointment | Other | Vehicle Ointment (Control) |
Primary outcome
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
Time frame: 12 weeks
Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
3 secondary outcomes
Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment
Time frame: 12 weeks
Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12
Time frame: 12 weeks
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12
Time frame: 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37170057Marathe K, Teng JMC, Guenthner S, Bunick CG, Kempers S, Eads K, Castelo-Soccio L, Mendelsohn AM, Raiz J, Murrell DF. Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study. Dermatol Ther (Heidelb). 2023 Jun;13(6):1255-1264. doi: 10.1007/s13555-023-00923-1. Epub 2023 May 11.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Isotretinoin in people with Congenital Ichthyosis, run by LEO Pharma at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Aug 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04154293, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.