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.
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
|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|