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Abstract
In 2 severe congenital ichthyosis (CI) subtypes, autosomal recessive lamellar ichthyosis (ARCI-LI) and X-linked recessive ichthyosis (XLRI), cutaneous manifestations include widespread scaling. Approved topical treatment options are limited to emollients and keratolytics.This analysis from the randomised Phase 2b CONTROL study assessed whether the efficacy and safety of TMB-001, a novel topical isotretinoin ointment formulation, differed between ARCI-LI and XLRI subtypes.Participants =9 years with genetically confirmed XLRI/ARCI-LI 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. Proportion of participants with =50% reduction vs baseline in VIIS scaling (VIIS-50; primary endpoint) and =2-grade reduction in Investigator Global Assessment (IGA)-scaling score vs baseline (key secondary endpoint) were evaluated. Adverse events (AEs) were monitored.Among enrolled participants (TMB-001 0.05% [n = 11], 0.1% [n = 10], and vehicle [n = 12]), 52% had ARCI-LI and 48% XLRI subtypes. Median age was 29 and 32 years for participants with ARCI-LI and XLRI, respectively. Overall, 33%/50%/17% of participants with ARCI-LI and 100%/33%/75% of participants with XLRI achieved VIIS-50, and improvement of =2-grade IGA score was observed in 33%/50%/0% of participants with ARCI-LI and 83%/33%/25% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively (nominal P = 0.026 for 0.05% vs vehicle; intent-to-treat population). Most AEs were application site reactions.Regardless of CI subtype, TMB-001 demonstrated greater proportions of participants achieving VIIS-50 and =2-grade IGA improvement vs vehicle.
View details for DOI 10.1093/ced/llad033
View details for PubMedID 36794376