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

• Sponsor: Aldeyra Therapeutics, Inc.
• Information provided by (Responsible Party): Aldeyra Therapeutics, Inc.

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Sjögren-Larsson Syndrome	Drug: Active topical NS2 1% dermatologic cream; Drug: Vehicle placebo 0.0% NS2 dermatologic cream	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 12 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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 Start Date: March 2015
• Actual Primary Completion Date: May 2016
• Actual Study Completion Date: June 2016

Arms and Interventions

Arm	Intervention/treatment
Experimental: Active topical NS2 1% dermatologic cream; NS2 1% topical cream for dermal application	Drug: Active topical NS2 1% dermatologic cream
Placebo Comparator: Topical vehicle dermatologic; Vehicle placebo for dermal application	Drug: Vehicle placebo 0.0% NS2 dermatologic cream

Outcome Measures

Primary Outcome Measures :

1. 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 Outcome Measures :

1. 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.

Other Outcome Measures:

1. Exploratory measurement of clinical presentation of ichthyosis pre and post-treatment [ Time Frame: Day 1, Week 4, Week 8 ]
   Change from baseline in intensity of ichthyosis symptoms in the treatment area pre and post-treatment.
2. Exploratory measurement of disease related biomarkers pre and post-treatment [ Time Frame: Day 1, Week 4, Week 8 ]
   Change from baseline in pre and post-treatment biomarkers.

Eligibility Criteria

• Ages Eligible for Study: 6 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

United States, Nebraska

University of Nebraska Medical Center

Omaha, Nebraska, United States, 68198

United States, Pennsylvania

Penn State Hershey Medical Center - Department of Dermatology

Hershey, Pennsylvania, United States, 17033

United States, Virginia

Pediatric and General Dermatology

Fairfax, Virginia, United States, 22031

Sponsors and Collaborators

Aldeyra Therapeutics, Inc.

Investigators

• Principal Investigator: William B. Rizzo, MD; University of Nebraska

More Information

• Responsible Party: Aldeyra Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT02402309 History of Changes
• Other Study ID Numbers: NS-003
• First Posted: March 30, 2015
• Last Update Posted: March 18, 2019
• Last Verified: August 2016

Keywords provided by Aldeyra Therapeutics, Inc.:

Sjögren-Larsson Syndrome; SLS; Ichthyosis; FALDH Deficiency; Neuro-ichthyosis; Fatty Alcohol:NAD+ Oxidoreductase Deficiency; Fatty Aldehyde Dehydrogenase Deficiency Disease; Congenital Icthyosis Mental Retardation Spasticity Syndrome; Spastic Neurologic Disorder; Oligophrenia

Additional relevant MeSH terms:

Sjogren's Syndrome; Ichthyosis; Sjogren-Larsson Syndrome; Syndrome; Disease; Pathologic Processes; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Abnormalities; Congenital Abnormalities; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn