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

Phase 2

Completed

• Conditions: Sjögren-Larsson Syndrome
• Interventions: Drug: Active topical NS2 1% dermatologic cream; Drug: Vehicle placebo 0.0% NS2 dermatologic cream

• Registration Number: NCT02402309
• Lead Sponsor: Aldeyra Therapeutics, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-03
• Study First Submitted: 2015-03-17
• Study First Submitted QC: 2015-03-25
• Study First Posted: 2015-03-30
• Primary Completion: 2016-05
• Study Completion: 2016-06
• Disposition First Submitted: 2019-03-13
• Disposition First Submitted QC: 2019-03-13
• Disposition First Posted: 2019-03-18
• Status Verified: 2022-11
• Results First Submitted: 2022-11-16
• Results First Submitted QC: 2022-11-16
• Last Update Submitted: 2022-11-16
• Results First Posted: 2022-12-13
• Last Update Posted: 2022-12-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Genetically-confirmed diagnosis of SLS
• Active ichthyosis on the lower extremities that is determined to be at least moderate severity

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Active topical NS2 1% dermatologic cream	Active topical NS2 1% dermatologic cream	NS2 1% topical cream for dermal application
Topical vehicle dermatologic	Vehicle placebo 0.0% NS2 dermatologic cream	Vehicle placebo for dermal application

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (3)

Penn State Hershey Medical Center - Department of Dermatology; 🇺🇸 Hershey, Pennsylvania, United States

Pediatric and General Dermatology; 🇺🇸 Fairfax, Virginia, United States

University of Nebraska Medical Center; 🇺🇸 Omaha, Nebraska, United States