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)
Overview
- Phase
- Phase 2
- Intervention
- Active topical NS2 1% dermatologic cream
- Conditions
- Sjögren-Larsson Syndrome
- Sponsor
- Aldeyra Therapeutics, Inc.
- Enrollment
- 12
- Locations
- 3
- Primary Endpoint
- Number of Participants Experiencing a Serious Adverse Event (SAE).
- Status
- Completed
- Last Updated
- 3 years ago
Overview
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.
Investigators
Eligibility Criteria
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
Arms & Interventions
Active topical NS2 1% dermatologic cream
NS2 1% topical cream for dermal application
Intervention: Active topical NS2 1% dermatologic cream
Topical vehicle dermatologic
Vehicle placebo for dermal application
Intervention: Vehicle placebo 0.0% NS2 dermatologic cream
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.