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

Primary Purpose

Sjögren-Larsson Syndrome

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Active topical NS2 1% dermatologic cream
Vehicle placebo 0.0% NS2 dermatologic cream
Sponsored by
Aldeyra Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sjögren-Larsson Syndrome focused on measuring 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

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • University of Nebraska Medical Center
  • Penn State Hershey Medical Center - Department of Dermatology
  • Pediatric and General Dermatology

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active topical NS2 1% dermatologic cream

Topical vehicle dermatologic

Arm Description

NS2 1% topical cream for dermal application

Vehicle placebo for dermal application

Outcomes

Primary Outcome Measures

Number of Participants Experiencing a Serious Adverse Event (SAE).
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.

Secondary Outcome Measures

Full Information

First Posted
March 17, 2015
Last Updated
November 16, 2022
Sponsor
Aldeyra Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02402309
Brief Title
A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
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 Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
March 2015 (undefined)
Primary Completion Date
May 2016 (Actual)
Study Completion Date
June 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aldeyra Therapeutics, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sjögren-Larsson Syndrome
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Active topical NS2 1% dermatologic cream
Arm Type
Experimental
Arm Description
NS2 1% topical cream for dermal application
Arm Title
Topical vehicle dermatologic
Arm Type
Placebo Comparator
Arm Description
Vehicle placebo for dermal application
Intervention Type
Drug
Intervention Name(s)
Active topical NS2 1% dermatologic cream
Intervention Type
Drug
Intervention Name(s)
Vehicle placebo 0.0% NS2 dermatologic cream
Primary Outcome Measure Information:
Title
Number of Participants Experiencing a Serious Adverse Event (SAE).
Time Frame
The safety assessment period is approximately 9 weeks.
Title
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
Time Frame
The safety assessment period is approximately 9 weeks.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William B. Rizzo, MD
Organizational Affiliation
University of Nebraska
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Penn State Hershey Medical Center - Department of Dermatology
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Pediatric and General Dermatology
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22031
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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