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

NCT ID: NCT02402309

Last Updated: 2022-12-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-03-31

Study Completion Date

2016-06-30

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.

Detailed Description

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Conditions

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Sjögren-Larsson Syndrome

Keywords

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Sjögren-Larsson Syndrome SLS 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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Active topical NS2 1% dermatologic cream

NS2 1% topical cream for dermal application

Group Type EXPERIMENTAL

Active topical NS2 1% dermatologic cream

Intervention Type DRUG

Topical vehicle dermatologic

Vehicle placebo for dermal application

Group Type PLACEBO_COMPARATOR

Vehicle placebo 0.0% NS2 dermatologic cream

Intervention Type DRUG

Interventions

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Active topical NS2 1% dermatologic cream

Intervention Type DRUG

Vehicle placebo 0.0% NS2 dermatologic cream

Intervention Type DRUG

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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Aldeyra Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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William B. Rizzo, MD

Role: PRINCIPAL_INVESTIGATOR

University of Nebraska

Locations

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University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Penn State Hershey Medical Center - Department of Dermatology

Hershey, Pennsylvania, United States

Site Status

Pediatric and General Dermatology

Fairfax, Virginia, United States

Site Status

Countries

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United States

Other Identifiers

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NS-003

Identifier Type: -

Identifier Source: org_study_id