Characteristics and outcomes for participants with congenital ichthyosis who responded to treatment with the topical isotretinoin formulation TMB-001: Results from the Phase 2b CONTROL study.

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Abstract

Emollients and keratolytics are frequently used to manage symptoms of congenital ichthyosis (CI). Systemic retinoid treatment is complicated by teratogenicity and dose-limiting adverse effects.This analysis from the randomised Phase 2b CONTROL study investigated the characteristics of participants who responded to treatment with TMB-001, a novel topical isotretinoin ointment formulation.Participants =9 years with genetically confirmed CI and =2 (out of 4) Visual Index for Ichthyosis Severity (VIIS) assessment areas with =3 scaling score were randomised 1:1:1 to TMB-001 0.05%:TMB-001 0.1%:vehicle twice daily for 12 weeks. Efficacy end points included the proportion of participants with =50% reduction vs baseline in VIIS-scaling (VIIS-50) and =2-grade reduction in Investigator Global Assessment (IGA)-scaling score vs baseline. Changes in body surface area (BSA) involvement, Dermatology Life Quality Index (DLQI) scores, and Itch-Numeric Rating Scale (I-NRS) scores were assessed.Of the 33 participants (TMB-001 0.05% [n?=?11], 0.1% [n?=?10], and vehicle [n?=?12]), median age was 29 years, and most were male (64%) and White (79%). Baseline demographics were generally similar among participants who did or did not achieve TMB-001 treatment success. Participants who had lower mean BSA involvement and higher DLQI and I-NRS scores at baseline were more likely to achieve VIIS-50. Similarly, higher baseline DLQI and I-NRS scores were associated with IGA response; BSA involvement was similar for IGA responders vs nonresponders.Higher DLQI and I-NRS scores at baseline were associated with participants achieving treatment success by VIIS-50 and IGA response. Lower BSA involvement was associated with VIIS-50 success.

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