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

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Sjögren-Larsson Syndrome

Intervention / Treatment
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* Drug: Active topical NS2 1% dermatologic cream
* Drug: Vehicle placebo 0.0% NS2 dermatologic cream


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Active topical NS2 1% dermatologic cream<br>NS2 1% topical cream for dermal application | Drug: Active topical NS2 1% dermatologic cream<br> <br> |
| Placebo Comparator: Topical vehicle dermatologic<br>Vehicle placebo for dermal application | Drug: Vehicle placebo 0.0% NS2 dermatologic cream<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Experiencing a Serious Adverse Event (SAE). |  | The safety assessment period is approximately 9 weeks. | 
| Number of Participants Experiencing an Adverse Event Leading to Discontinuation. |  | The safety assessment period is approximately 9 weeks. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SLS, Sjögren-Larsson Syndrome, FALDH Deficiency, Neuro-ichthyosis, Fatty Alcohol:NAD+ Oxidoreductase Deficiency, Fatty Aldehyde Dehydrogenase Deficiency Disease, Congenital Icthyosis Mental Retardation Spasticity Syndrome, Spastic Neurologic Disorder, Oligophrenia, Ichthyosis