A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)

Aldeyra Therapeutics, Inc.12 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Sjögren-Larsson Syndrome

Interventions

Active topical NS2 1% dermatologic creamDRUG

Vehicle placebo 0.0% NS2 dermatologic creamDRUG

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Genetically-confirmed diagnosis of SLS * Active ichthyosis on the lower extremities that is determined to be at least moderate severity Exclusion Criteria: * Evidence of an active infection * Currently receiving immunosuppressive therapy, including intermittent or low-dose corticosteroids and is not able or willing to suspend from 2 weeks before and during the study * Currently receiving systemic or topical retinoids, other topically applied drugs, or other supplements that could interfere with dermatologic examination findings * Received an investigational systemic or topically administered drug within 30 days before screening

Locations (3)

University of Nebraska Medical Center

Omaha, Nebraska, United States

Penn State Hershey Medical Center - Department of Dermatology

Hershey, Pennsylvania, United States

Pediatric and General Dermatology

Fairfax, Virginia, United States

Outcomes

Primary Outcomes

Number of Participants Experiencing a Serious Adverse Event (SAE).

Time frame: The safety assessment period is approximately 9 weeks.

Number of Participants Experiencing an Adverse Event Leading to Discontinuation.

Time frame: The safety assessment period is approximately 9 weeks.

Data from ClinicalTrials.gov

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