Phase 2
N=34
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
Congenital Ichthyosis
Bottom Line
View on ClinicalTrials.gov: NCT04154293 ↗Enrolled (actual)
34
Serious AEs
0.0%
Results posted
Aug 2022
Primary outcome: Primary: Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement — 4; 7; 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Isotretinoin (Drug); Vehicle (Other)
- Age
- Pediatric, Adult, Older Adult · 9+ yrs
- Sex
- All
- Sponsor
- LEO Pharma
- Primary completion
- Aug 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement |
4; 7; 4 | — |
| SECONDARY Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment |
1; 6; 4 | — |
| SECONDARY Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12 |
-1.7; -1.9; 1.1 | — |
| SECONDARY Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12 |
-3.4; -3.2; -1.3 | — |
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 (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.
Funding Source FDA-OOPD
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT04154293). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.