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
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
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
- 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.
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02402309
|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|
|Principal Investigator:||William B. Rizzo, MD||University of Nebraska|