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

Aldeyra Therapeutics

Status and phase: Completed; Phase 2

Conditions: Sjögren-Larsson Syndrome

Treatments: Drug: Active topical NS2 1% dermatologic cream; Drug: Vehicle placebo 0.0% NS2 dermatologic cream

Study type: Interventional

Funder types: Industry

Identifiers

NCT02402309

NS-003

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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12 participants in 2 patient groups, including a placebo group

Active topical NS2 1% dermatologic cream

Experimental group

Description:

NS2 1% topical cream for dermal application

Treatment:

Drug: Active topical NS2 1% dermatologic cream

Topical vehicle dermatologic

Placebo Comparator group

Description:

Vehicle placebo for dermal application

Treatment:

Drug: Vehicle placebo 0.0% NS2 dermatologic cream