Conditions: Sjögren-Larsson Syndrome
Keywords: Sjögren-Larsson Syndrome SLS FALDH Deficiency Neuro-ichthyosis Fatty Alcohol:NAD+ Oxidoreductase Deficiency Fatty Aldehyde Dehydrogenase Deficiency Disease Congenital Icthyosis Mental Retardation Spasticity Syndrome Spastic Neurologic Disorder Oligophrenia Ichthyosis
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 12
Healthy volunteers: no

Brief summary:
This is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, pharmacokinetic (PK), and exploratory activity of topically-applied NS2 dermatologic cream administered once-daily (QD) to subjects with ichthyosis secondary to Sjögren- Larsson Syndrome (SLS).

NS2 is expected to trap fatty aldehydes that are pathogenic in SLS patients, and thereby diminish the lipid-aldehyde adduct formation that likely results in ichthyosis associated with SLS, and potentially reduce the mild dermal inflammation characteristic of SLS.

Detailed description:

Arms:
arm 1: NS2 1% topical cream for dermal application arm 2: Vehicle placebo for dermal application

Interventions: intervention 1: Active topical NS2 1% dermatologic cream intervention 2: Vehicle placebo 0.0% NS2 dermatologic cream
Intervention descriptions: intervention 1: None intervention 2: None

Protocol document: