This is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, 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.
Full Title of Study: “Phase II Study of the Safety, Pharmacokinetics, and Exploratory Activity of Once Daily (QD) Topical Application of NS2 Cream to Treat Ichthyosis in Subjects With Sjögren-Larsson Syndrome (SLS)”
- Study Type: Interventional
- Study Design
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Primary Purpose: Treatment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Study Primary Completion Date: May 2016
- Drug: Active topical NS2 1% dermatologic cream
- Drug: Vehicle placebo 0.0% NS2 dermatologic cream
Arms, Groups and Cohorts
- Experimental: Active topical NS2 1% dermatologic cream
- NS2 1% topical cream for dermal application
- Placebo Comparator: Topical vehicle dermatologic
- Vehicle placebo for dermal application
Clinical Trial Outcome Measures
- Number of Participants with Adverse Events as a measure of Safety and Tolerability
- Time Frame: Week 8
- As this is an early-stage safety, tolerability and exploratory activity study, no specific endpoint(s) are designated as primary or secondary.
- Pharmacokinetics – Peak Plasma Concentration (Cmax)
- Time Frame: Day 1, Day 2, Week 4, Week 8
- Determination of single and repeat-dose PK profile of 1% NS2 topically administered to the skin.
Participating in This Clinical Trial
- Genetically-confirmed diagnosis of SLS
- Active ichthyosis on the lower extremities that is determined to be at least moderate severity
- 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
Gender Eligibility: All
Minimum Age: 6 Years
Maximum Age: N/A
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Aldeyra Therapeutics, Inc.
- Provider of Information About this Clinical Study
- Overall Official(s)
- William B. Rizzo, MD, Principal Investigator, University of Nebraska
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