A Phase 3 interventional study of TMB-001 and Matching Vehicle in Ichthyosis, sponsored by LEO Pharma. Completed at 35 sites in 5 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-05-22.
Sponsored by LEO Pharma · Phase 3, Interventional, and Treatment
This is a randomized, double-blind and vehicle-controlled Phase III study to evaluate the safety and efficacy of topical TMB-001 0.05% ointment for the treatment of congenital ichthyosis (CI) in subjects with either the RXLI or ARCI subtypes.
In addition, a subset of preselected centers will recruit subjects in parallel with either the RXLI or ARCI subtypes for enrollment into an Optional Maximal Use arm for evaluation of the systemic exposure and safety of topical TMB-001 0.05% ointment for the treatment of CI.
The Phase III Study is designed in three periods:
- Period 1 - Induction (3 weeks):
At the beginning of the 3-week Induction Period, eligible subjects will be randomized (2:1 ratio) to either TMB-001 0.05% once-a-day (QD) or Vehicle QD treatment, with use of mandatory standardized bland emollient (Cetaphil™) provided by the Sponsor.
- Period 2 - Treatment (9 weeks):
The dosing frequency in the 9-week treatment period will be increased in each treatment group to TMB-001 0.05% BID or Vehicle BID. Mandatory bland emollient will be discontinued.
- Period 3 - Maintenance (12 weeks):
At Week 12, eligible subjects in the TMB-001 treatment group will be randomized (1:1 ratio) to an open-label treatment with TMB-001 0.05% BID or TMB-001 0.05% QD. To be eligible, subjects must have achieved a ≥1-point reduction in IGA score from Baseline. Subjects with less than a 1-point reduction in IGA score from Baseline will be discontinued from the study.
Vehicle-treated subjects who achieved \<1-point reduction in IGA score from Baseline are eligible to cross over to the TMB-001 0.05% BID treatment group. Subjects with a ≥1-point reduction in IGA score from Baseline on vehicle will be discontinued from the study.
Subjects at the end of the study or subjects discontinued from the study at any time will be followed-up for additional 2 weeks for AEs.
This is a multicenter, randomized, double-blind, vehicle-controlled Phase III study to evaluate the efficacy and safety of TMB-001 0.05% topical ointment in the treatment of CI. Subjects will be selected according to predefined entry criteria. The study treatment duration is 24 weeks and expected to be sufficient to show a treatment effect.
Isotretinoin is an approved active pharmacological ingredient with a long history of safe use in humans. However, isotretinoin is a known teratogen with an extremely high risk for severe birth defects if pregnancy occur while taking oral isotretinoin in any amount, even for a short period of time. Therefore oral, systemic isotretinoin requires an iPLEDGE program (iPLEDGE 2012), which is a risk management distribution program mandated by the FDA. To minimize pregnancy risks, women of childbearing potential (WOCBP) will only be enrolled if they agree to use highly effective methods of contraception consistently and correctly and undergo regular pregnancy testing.
To minimize bias, subjects will be blinded and randomly assigned to treatment with TMB-001 0.05% or Vehicle, additionally subjects who had previously been treated with TMB 001 will be excluded from this study but can be enrolled in the optional Maximal Use arm (in a subset of preselected centers). The use of a vehicle control group is consistent with FDA's standard for generating valid scientific evidence to definitively support safety and efficacy. The vehicle group accounts for the effects of treatment that do not depend on the test treatment. The study is designed to mitigate safety risks by using an initial 2:1 randomization (active treatment to vehicle), along with frequent clinic visits over the 12-week treatment period. The subsequent 1:1 randomization of eligible TMB-001 0.05% treated subjects to two dosing regimen (QD or BID) allows for assessment of the optimal TMB-001 0.05% maintenance therapy as well as provides additional safety data for 12 weeks. The cross-over of eligible subjects from the vehicle control group will also provide additional safety data.
Overall, the study design is considered to be scientifically robust and clinically relevant for evaluating TMB-001 0.05% treatment for the safe and effective treatment of CI.
33 studies on the registry are indexed under Ichthyosis; 11 are open to participants now.
This study's enrollment of 153 is above the median of 26 across 23 interventional studies indexed under Ichthyosis.
Browse Ichthyosis studies →LEO Pharma is the lead sponsor of 225 studies on the registry; 5 are open to participants now.
Of its 32 completed or terminated interventional studies of FDA-regulated products, 24 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
The amount of CI affected skin in the Treatment Area at Baseline will be between a minimum of 10% and maximum of 90% of the total BSA (1% BSA is approximately equal to the surface area of the subject's palm and fingers, with the fingers extended yet grouped together, creating a flat oval-like surface area).
Documented history of moderate to severe disease at Screening. Subject's designated VIIS Assessment Areas at Baseline (not applicable for Optional Maximal Use arm):
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TMB-001 0.05% ointment: Induction phase QD for 3 weeks, followed by BID for 9 weeks. Maintenance therapy for additional 12 weeks randomized QD vs BID
Drug: TMB-001
Matching vehicle ointment: Induction phase QD for 3 weeks, followed by BID for 9 weeks. Cross over to 12 weeks TMB-001 0.05% BID.
Drug: Matching Vehicle
Optional Parallel Arm to evaluate the systemic exposure and safety of TMB-001 0.05% under conditions of maximal use.
Drug: TMB-001
Topical TMB-001 0.05% QD/BID
Topical Vehicle
Change in Investigator Global Assessment (IGA) Score
Comparison of proportions of subjects in percentages with ≥2-point changes from Baseline in Investigator Global Assessment (IGA)-scaling and fissuring scores in the Treatment Area at Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
Time frame: 12 weeks
Number of Subjects With IGA Scores
Comparison of proportion of subjects in percentages with IGA scores of clear or almost clear at Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
Time frame: 12 weeks
Change in IGA-scaling Severity Sub-score
Comparison of proportion of subjects in percentages who achieve IGA-scaling severity sub-score improvement ≥ 2-points from Baseline to Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
Time frame: 12 weeks
Change in Worst Itch-Quality of Life (QoL) Scores
Comparison of proportion of subjects in percentages with ≥4-point improvement from baseline in Worst Itch-QoL scores at Week 12 in subjects with baseline WI-NRS of ≥7 between TMB-001 0.05% and vehicle-treated subjects. Itch-Numeric Rating Scale (I-NRS) and Worst Itch-Numeric Rating Scale (WI-NRS) is an 0-10 scale where 0 is "no itching" and 10 is "worst itch imaginable".
Time frame: 12 weeks
Change in Visual Index of Ichthyosis Severity (VIIS) Score
Comparison of proportion of subjects in percentages who achieve 50% reduction from Baseline in VIIS-scaling scores at Week 12 in all areas with Baseline VIIS score ≥3 between TMB-001 0.05% and vehicle-treated subjects. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
Time frame: 12 weeks
Change in VIIS Score
Comparison of proportion of subjects in percentages who achieve 25% reduction from Baseline in VIIS-scaling scores at Week 12 in all areas with Baseline VIIS score ≥3 between TMB-001 0.05% and vehicle-treated subjects. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
Time frame: 12 weeks
Change in IGA-fissuring Severity Sub-scores
Comparison of proportion of subjects in percentages achieving ≥2 point improvement in IGA-fissuring severity sub-scores from Baseline to Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
Time frame: 12 weeks
Change in IGA Score
Comparison of proportions of subjects in percentages achieving ≥2-point improvement from Baseline in IGA scores at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
Time frame: 24 weeks
Change in VIIS Score
Comparison of proportion of subjects in percentages who achieve 50% reduction from Baseline in VIIS-scaling scores at Week 24 in all areas with Baseline VIIS score ≥3 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
Time frame: 24 weeks
Change in Ichthyosis Quality of Life (IQoL)-32 Scores
Comparison of proportions of subjects in percentages with ≥11-point changes from Baseline in IQOL-32 scores at Week 12 between TMB-001 0.05% and vehicle-treated subjects. The IQoL-32 is a questionnaire containing 32, each scored from 0 to 4 ('not applicable', 'not at all', 'a little', 'a lot', 'tremendously') for a total score that varies between 0 and 128. A higher score, the higher impact on quality of life.
Time frame: 12 weeks
Change in Dermatology Life Quality Index (DLQI) Scores
Comparison of proportion of subjects in percentages with reduction from Baseline in DLQI ≥4 points at Week 12 between TMB-001 0.05% and vehicle-treated subjects in adult subjects with Baseline scores ≥11. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
Time frame: 12 weeks
Change in Children's Dermatology Life Quality Index (CDLQI) Scores
Comparison of proportion of pediatric subjects in percentages with reduction from Baseline in CDLQI ≥4 points at Week 12 between TMB-001 0.05% and vehicle-treated subjects in pediatric subjects with Baseline scores of ≥13. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
Time frame: 12 weeks
Change in Itch-Quality of Life Scores - I-NRS
Comparison of proportions of subjects in percentages with I-NRS improvement ≥4 points from Baseline in Itch-QoL scores (in subjects with Baseline I-NRS ≥7) at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
Time frame: 24 weeks
Change in Itch-Quality of Life Scores - WI-NRS
Comparison of proportions of subjects in percentages with WI-NRS improvement ≥4 points from Baseline in Itch-QoL scores (in subjects with Baseline WI-NRS ≥7) at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
Time frame: 24 weeks
Change in DLQI Scores
Comparison of proportions of adult subjects in percentages with DLQI changes of ≥4-point from Baseline scores (in subjects with Baseline scores ≥11) at Week 24 randomized to TMB-001 0.05% BID and QD maintenance dosing. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
Time frame: 24 weeks
Change in CDLQI Scores
Comparison of proportions of pediatric subjects in percentages with CDLQI changes of ≥4-point from Baseline scores (in subjects with Baseline scores ≥13) at Week 24 randomized to TMB-001 0.05% BID and QD maintenance dosing. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
Time frame: 24 weeks
Change in IQoL-32 Scores
Proportions of subjects in percentages with ≥11-point change from Baseline in IQoL-32 at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing in adult subjects. The IQoL-32 is a questionnaire containing 32, each scored from 0 to 4 ('not applicable', 'not at all', 'a little', 'a lot', 'tremendously') for a total score that varies between 0 and 128. A higher score, the higher impact on quality of life.
Time frame: 24 weeks
To Investigate the Proportion of Subjects Experiencing Local Skin Reactions (LSRs) With Topically Applied TMB-001 0.05% Ointment.
Comparison of proportion of subjects in percentages experiencing LSRs through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
Time frame: 12 weeks
To Investigate the Proportion of Subjects Experiencing Treatment-emergent Adverse Events (TEAEs)
Comparison of proportion of subjects in percentages experiencing TEAEs through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
Time frame: 12 weeks
To Investigate the Proportion of Subjects Experiencing LSRs With Topically Applied TMB-001 0.05% Ointment.
Comparison of proportion of subjects in percentages experiencing LSRs through Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
Time frame: 24 weeks
To Investigate the Proportion of Subjects Experiencing TEAEs
Comparison of proportion of subjects in percentages experiencing TEAEs through Week 24.
Time frame: 24 weeks
To Investigate the Proportion of Subjects Demonstrating Clinically Confirmed Allergic Contact Dermatitis
Comparison of proportion of subjects in percentages demonstrating clinically confirmed allergic contact dermatitis by patch testing through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
Time frame: Through week 12
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - Cmax After Multiple Dosing - Adults
maximal observed plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 14 days
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - Cmax After Multiple Dosing - Adolescents
maximal observed plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 14 days
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - AUC0-24 After Multiple Dosing - Adults
AUC0-24 = area under the curve over the first 24 hours post dose. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 0-24hrs after dose
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - AUC0-24 After Multiple Dosing - Adolescents
AUC0-24 = area under the curve over the first 24 hours post dose. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 0-24hrs after dose
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - Tmax After Multiple Dosing - Adults
Tmax = time to maximal plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 14 days
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - Tmax After Multiple Dosing - Adolescents
Tmax = time to maximal plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 14 days
Maximal Use Arm: Isotretinoin, Tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin - Steady State Concentration After Multiple Dosing - Children
Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
Time frame: 14 days
Maximal Use Arm: Safety and Tolerability - LSRs
Local skin reactions (burnings/stinging, erythema, erosions and edema) are reported as LSRs.
Time frame: 12 weeks
Maximal Use Arm: Safety and Tolerability - TEAEs
Local safety are reported as severe TEAEs related to treatment area.
Time frame: 12 weeks
The study was conducted in 34 sites across the United States, Canada, Germany, France, and Italy enrolled subjects, and 33 of these sites randomized subjects.
| Milestone | TMB-001 0.05% | Vehicle | TMB-001 0.05% QD | TMB-001 0.05% BID | Maximal Use |
|---|---|---|---|---|---|
| Started | 79 | 40 | 0 | 0 | 34 |
| Dosed | 78 | 38 | 0 | 0 | 34 |
| Completed | 65 | 36 | 0 | 0 | 32 |
| Not completed | 14 | 4 | 0 | 0 | 2 |
| Withdrew: Various reasons | 14 | 4 | 0 | 0 | 2 |
| Milestone | TMB-001 0.05% | Vehicle | TMB-001 0.05% QD | TMB-001 0.05% BID | Maximal Use |
|---|---|---|---|---|---|
| Started | 0 | 0 | 28 | 38 | 32 |
| Completed | 0 | 0 | 28 | 37 | 31 |
| Not completed | 0 | 0 | 0 | 1 | 1 |
| Withdrew: Various reasons | 0 | 0 | 0 | 1 | 1 |
Comparison of proportions of subjects in percentages with ≥2-point changes from Baseline in Investigator Global Assessment (IGA)-scaling and fissuring scores in the Treatment Area at Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Investigator Global Assessment (IGA) Score | 41.4 | 44.9 |
Comparison of proportion of subjects in percentages with IGA scores of clear or almost clear at Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Number of Subjects With IGA Scores | 35.8 | 38.9 |
Comparison of proportion of subjects in percentages who achieve IGA-scaling severity sub-score improvement ≥ 2-points from Baseline to Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in IGA-scaling Severity Sub-score | 40.4 | 36.4 |
Comparison of proportion of subjects in percentages with ≥4-point improvement from baseline in Worst Itch-QoL scores at Week 12 in subjects with baseline WI-NRS of ≥7 between TMB-001 0.05% and vehicle-treated subjects. Itch-Numeric Rating Scale (I-NRS) and Worst Itch-Numeric Rating Scale (WI-NRS) is an 0-10 scale where 0 is "no itching" and 10 is "worst itch imaginable".
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Worst Itch-Quality of Life (QoL) Scores | 54.4 | 68.7 |
Comparison of proportion of subjects in percentages who achieve 50% reduction from Baseline in VIIS-scaling scores at Week 12 in all areas with Baseline VIIS score ≥3 between TMB-001 0.05% and vehicle-treated subjects. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Visual Index of Ichthyosis Severity (VIIS) Score | 47.2 | 42.7 |
Comparison of proportion of subjects in percentages who achieve 25% reduction from Baseline in VIIS-scaling scores at Week 12 in all areas with Baseline VIIS score ≥3 between TMB-001 0.05% and vehicle-treated subjects. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in VIIS Score | 71.3 | 64.1 |
Comparison of proportion of subjects in percentages achieving ≥2 point improvement in IGA-fissuring severity sub-scores from Baseline to Week 12 between TMB-001 0.05% and vehicle-treated subjects. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in IGA-fissuring Severity Sub-scores | 54.1 | 57.9 |
Comparison of proportions of subjects in percentages achieving ≥2-point improvement from Baseline in IGA scores at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing. Investigator's Global Assessment Score is a 0-4 scale, where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in IGA Score | 57.1 (38.8 to 75.5) | 85.2 (71.8 to 98.6) |
Comparison of proportion of subjects in percentages who achieve 50% reduction from Baseline in VIIS-scaling scores at Week 24 in all areas with Baseline VIIS score ≥3 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing. Visual Index for Ichthyosis Severity Score is a 0-4 scale where 0 is clear and 4 is severe.
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in VIIS Score | 63.0 (44.7 to 81.2) | 85.2 (71.8 to 98.6) |
Comparison of proportions of subjects in percentages with ≥11-point changes from Baseline in IQOL-32 scores at Week 12 between TMB-001 0.05% and vehicle-treated subjects. The IQoL-32 is a questionnaire containing 32, each scored from 0 to 4 ('not applicable', 'not at all', 'a little', 'a lot', 'tremendously') for a total score that varies between 0 and 128. A higher score, the higher impact on quality of life.
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Ichthyosis Quality of Life (IQoL)-32 Scores | 47.1 | 9.5 |
Comparison of proportion of subjects in percentages with reduction from Baseline in DLQI ≥4 points at Week 12 between TMB-001 0.05% and vehicle-treated subjects in adult subjects with Baseline scores ≥11. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Dermatology Life Quality Index (DLQI) Scores | 86.7 | 37.5 |
Comparison of proportion of pediatric subjects in percentages with reduction from Baseline in CDLQI ≥4 points at Week 12 between TMB-001 0.05% and vehicle-treated subjects in pediatric subjects with Baseline scores of ≥13. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
| percentage of responders | TMB-001 0.05% | Vehicle |
|---|---|---|
| Change in Children's Dermatology Life Quality Index (CDLQI) Scores | 80.0 | — |
Comparison of proportions of subjects in percentages with I-NRS improvement ≥4 points from Baseline in Itch-QoL scores (in subjects with Baseline I-NRS ≥7) at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in Itch-Quality of Life Scores - I-NRS | — | 100 (100.0 to 100.0) |
Comparison of proportions of subjects in percentages with WI-NRS improvement ≥4 points from Baseline in Itch-QoL scores (in subjects with Baseline WI-NRS ≥7) at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in Itch-Quality of Life Scores - WI-NRS | 66.7 (0.00 to 100.0) | 75.0 (19.2 to 100.0) |
Comparison of proportions of adult subjects in percentages with DLQI changes of ≥4-point from Baseline scores (in subjects with Baseline scores ≥11) at Week 24 randomized to TMB-001 0.05% BID and QD maintenance dosing. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in DLQI Scores | 75.0 (32.6 to 100.0) | 100 (100.0 to 100.0) |
Comparison of proportions of pediatric subjects in percentages with CDLQI changes of ≥4-point from Baseline scores (in subjects with Baseline scores ≥13) at Week 24 randomized to TMB-001 0.05% BID and QD maintenance dosing. The age-appropriate questionnaire contains 10 questions scored from 0 (no impact of skin disease on QoL) to 30 (maximum impact on QoL).
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in CDLQI Scores | 100 (100.0 to 100.0) | 100 (100.0 to 100.0) |
Proportions of subjects in percentages with ≥11-point change from Baseline in IQoL-32 at Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing in adult subjects. The IQoL-32 is a questionnaire containing 32, each scored from 0 to 4 ('not applicable', 'not at all', 'a little', 'a lot', 'tremendously') for a total score that varies between 0 and 128. A higher score, the higher impact on quality of life.
| percentage of responders | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| Change in IQoL-32 Scores | 40.0 (9.6 to 70.4) | 60.0 (29.6 to 90.4) |
Comparison of proportion of subjects in percentages experiencing LSRs through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
| percentage of participants | TMB-001 0.05% | Vehicle |
|---|---|---|
| To Investigate the Proportion of Subjects Experiencing Local Skin Reactions (LSRs) With Topically Applied TMB-001 0.05% Ointment. | 64.1 (52.4 to 74.7) | 36.8 (21.8 to 54.0) |
Comparison of proportion of subjects in percentages experiencing TEAEs through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
| percentage of participants | TMB-001 0.05% | Vehicle |
|---|---|---|
| To Investigate the Proportion of Subjects Experiencing Treatment-emergent Adverse Events (TEAEs) | 76.9 | 63.2 |
Comparison of proportion of subjects in percentages experiencing LSRs through Week 24 between subjects randomized to TMB-001 0.05% BID and QD maintenance dosing.
| percentage of participants | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| To Investigate the Proportion of Subjects Experiencing LSRs With Topically Applied TMB-001 0.05% Ointment. | 32.1 (15.9 to 52.4) | 40.7 (22.4 to 61.2) |
Comparison of proportion of subjects in percentages experiencing TEAEs through Week 24.
| percentage of participants | TMB-001 0.05% QD | TMB-001 0.05% BID |
|---|---|---|
| To Investigate the Proportion of Subjects Experiencing TEAEs | 53.6 (33.9 to 72.5) | 48.1 (28.7 to 68.1) |
Comparison of proportion of subjects in percentages demonstrating clinically confirmed allergic contact dermatitis by patch testing through Week 12 between TMB-001 0.05% and vehicle-treated subjects.
| percentage of participants | TMB-001 0.05% | Vehicle |
|---|---|---|
| To Investigate the Proportion of Subjects Demonstrating Clinically Confirmed Allergic Contact Dermatitis | 0 | 0 |
maximal observed plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| ng/mL | Maximal Use |
|---|---|
| Isotretinoin | 4.13 ± 6.48 |
| 4-Oxo-Isotretinoin | 16.7 ± 26 |
| Tretinoin | 0.04 ± 0.12 |
| 4-oxo-tretinoin | 0.00 ± 0.00 |
maximal observed plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| ng/mL | Maximal Use |
|---|---|
| Isotretinoin | 7.7 ± 1.17 |
| 4-Oxo-Isotretinoin | 25.44 ± 10.11 |
| Tretinoin | 4.52 ± 6.39 |
| 4-oxo-tretinoin | 3.60 ± 5.08 |
AUC0-24 = area under the curve over the first 24 hours post dose. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| hr*ng/mL | Maximal Use |
|---|---|
| Isotretinoin | 78.44 ± 122.08 |
| 4-Oxo-Isotretinoin | 352.95 ± 577.05 |
| Tretinoin | 0.17 ± 0.54 |
| 4-oxo-tretinoin | 0.00 ± 0.00 |
AUC0-24 = area under the curve over the first 24 hours post dose. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| hr*ng/mL | Maximal Use |
|---|---|
| Isotretinoin | 101.34 ± 61.15 |
| 4-Oxo-Isotretinoin | 432.96 ± 121.81 |
| Tretinoin | 13.74 ± 19.43 |
| 4-oxo-tretinoin | 10.15 ± 14.35 |
Tmax = time to maximal plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| hours | Maximal Use |
|---|---|
| Isotretinoin | 1.50 (0 to 24) |
| 4-Oxo-Isotretinoin | 5 (0 to 24) |
| Tretinoin | 0.00 (0.00 to 6.00) |
| 4-oxo-tretinoin | 0.00 (0.00 to 0.00) |
Tmax = time to maximal plasma concentration. \> Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| hours | Maximal Use |
|---|---|
| Isotretinoin | 14 (4 to 24) |
| 4-Oxo-Isotretinoin | 18 (12 to 24) |
| Tretinoin | 2.00 (0.00 to 4.00) |
| 4-oxo-tretinoin | 1.00 (0.00 to 2.00) |
Assessment of concentrations of isotretinoin, tretinoin, 4-oxo-tretinoin and 4-oxo-isotretinoin.
| ng/mL | Maximal Use |
|---|---|
| Isotretinoin | 1.27 ± 1.2 |
| 4-Oxo-Isotretinoin | 9.53 ± 6.13 |
| Tretinoin | 0 ± 0 |
| 4-oxo-tretinoin | 0 ± 0 |
Local skin reactions (burnings/stinging, erythema, erosions and edema) are reported as LSRs.
| events | Maximal Use |
|---|---|
| Erythema | 16 |
| Erosions | 7 |
| Edema | 2 |
| Burning/stinging | 17 |
Local safety are reported as severe TEAEs related to treatment area.
| events | Maximal Use |
|---|---|
| Pruritus | 2 |
| Administration site reactions | 8 |
Collected over 24 weeks for the phase 3 trial part and 12 weeks for the Maximal use arm. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Double-blinded Phase: TMB-001 0.05% | 0/78 (0%) | 1/78 (1.3%) | 47/78 (60.3%) |
| Double-blinded Phase: Vehicle | 0/38 (0%) | 0/38 (0%) | 17/38 (44.7%) |
| Open-label Phase: TMB-001 0.05% QD | 0/28 (0%) | 0/28 (0%) | 11/28 (39.3%) |
| Open-label Phase: TMB-001 0.05% BID | 0/38 (0%) | 0/38 (0%) | 14/38 (36.8%) |
| Maximal Use | 0/34 (0%) | 0/34 (0%) | 22/34 (64.7%) |
| Event | Double-blinded Phase: TMB-001 0.05% | Double-blinded Phase: Vehicle | Open-label Phase: TMB-001 0.05% QD | Open-label Phase: TMB-001 0.05% BID | Maximal Use |
|---|---|---|---|---|---|
| Viral rashInfections and infestations | 1/78 | — | — | — | — |
| Event | Double-blinded Phase: TMB-001 0.05% | Double-blinded Phase: Vehicle | Open-label Phase: TMB-001 0.05% QD | Open-label Phase: TMB-001 0.05% BID | Maximal Use |
|---|---|---|---|---|---|
| Application site painGeneral disorders | 23/78 | 5/38 | 4/28 | 3/38 | 13/34 |
| Application site erythemaGeneral disorders | 27/78 | 4/38 | 5/28 | 6/38 | 11/34 |
| Application site erosionGeneral disorders | 14/78 | 2/38 | 0/28 | 4/38 | 7/34 |
| Application site pruritusGeneral disorders | 12/78 | 0/38 | 0/28 | 5/38 | 5/34 |
| HeadacheNervous system disorders | 7/78 | 1/38 | 0/28 | 3/38 | 1/34 |
| NasopharyngitisInfections and infestations | 4/78 | 3/38 | 2/28 | 1/38 | 1/34 |
| Application site oedemaGeneral disorders | 6/78 | 1/38 | 0/28 | 1/38 | 2/34 |
| ErythemaSkin and subcutaneous tissue disorders | 6/78 | 0/38 | 0/28 | 0/38 | 0/34 |
| PyrexiaGeneral disorders | 3/78 | 1/38 | 1/28 | 0/38 | 2/34 |
| PruritusSkin and subcutaneous tissue disorders | 3/78 | 2/38 | 0/28 | 0/38 | 2/34 |
TMB-001 0.05% and vehicle: Intent-to-Treat (ITT) population = all randomized subjects who received at least one dose of study medication. \> Maximal Use = all subjects enrolled in the maximal use arm who receive at least 1 dose of study medication.
| Age, Customized(Participants) | TMB-001 0.05% | Vehicle | Maximal Use | Total |
|---|---|---|---|---|
| Age — 6-11 years | 16 | 8 | 9 | 33 |
| Age — 12-16 years | 19 | 8 | 7 | 34 |
| Age — >=17 years | 43 | 22 | 18 | 83 |
| Sex: Female, Male(Participants) | TMB-001 0.05% | Vehicle | Maximal Use | Total |
|---|---|---|---|---|
| Female | 29 | 15 | 16 | 60 |
| Male | 49 | 23 | 18 | 90 |
| Ethnicity (NIH/OMB)(Participants) | TMB-001 0.05% | Vehicle | Maximal Use | Total |
|---|---|---|---|---|
| Hispanic or Latino | 2 | 2 | 2 | 6 |
| Not Hispanic or Latino | 28 | 19 | 25 | 72 |
| Unknown or Not Reported | 48 | 17 | 7 | 72 |
| Race (NIH/OMB)(Participants) | TMB-001 0.05% | Vehicle | Maximal Use | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 1 | 1 |
| Asian | 3 | 2 | 3 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 1 | 0 | 1 |
| Black or African American | 2 | 5 | 7 | 14 |
| White | 57 | 25 | 21 | 103 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 16 | 5 | 2 | 23 |
| Body Mass Index (BMI)(kg/m^2) | TMB-001 0.05% | Vehicle | Maximal Use | Total |
|---|---|---|---|---|
| Mean | 22.903 ± 5.4218 | 24.092 ± 7.6000 | 24.468 ± 8.6960 | 23.559 ± 6.8388 |
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