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

Overview

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

  • 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

Interventions

  • 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

Primary 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.

Secondary Measures

  • 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

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

Gender Eligibility: All

Minimum Age: 6 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Aldeyra Therapeutics, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • William B. Rizzo, MD, Principal Investigator, University of Nebraska

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