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

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ClinicalTrials.gov Identifier: NCT02402309
Recruitment Status : Completed
First Posted : March 30, 2015
Last Update Posted : March 18, 2019
Sponsor:
Information provided by (Responsible Party):
Aldeyra Therapeutics, Inc.

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

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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


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



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.



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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02402309


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

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Responsible Party: Aldeyra Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02402309     History of Changes
Other Study ID Numbers: NS-003
First Posted: March 30, 2015    Key Record Dates
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:
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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