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CompletedNCT02402309Updated Dec 13, 2022Results posted

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

A Phase 2 interventional study of Active topical NS2 1% dermatologic cream and Vehicle placebo 0.0% NS2 dermatologic cream in Sjögren-Larsson Syndrome, sponsored by Aldeyra Therapeutics, Inc.. Completed at 3 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2022-12-13.

Sponsored by Aldeyra Therapeutics, Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
12
Allocation
Randomized
Ages
6 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • 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
03

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

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

Exclusion criteria

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
12 participants (actual)

Study arms

  • Experimental
    Active topical NS2 1% dermatologic cream

    NS2 1% topical cream for dermal application

    Drug: Active topical NS2 1% dermatologic cream

  • Placebo comparator
    Topical vehicle dermatologic

    Vehicle placebo for dermal application

    Drug: Vehicle placebo 0.0% NS2 dermatologic cream

Interventions

  • DrugActive topical NS2 1% dermatologic cream
  • DrugVehicle placebo 0.0% NS2 dermatologic cream
05

What researchers measure

Primary outcomes

  1. Number of Participants Experiencing a Serious Adverse Event (SAE).

    Time frame: The safety assessment period is approximately 9 weeks.

  2. Number of Participants Experiencing an Adverse Event Leading to Discontinuation.

    Time frame: The safety assessment period is approximately 9 weeks.

06

Results

Posted Dec 13, 2022

Participant flow

Participant flow — Overall Study
MilestoneTopical NS2 1% Dermatologic CreamTopical Vehicle Dermatologic
Started66
Completed66
Not completed00

Outcome measures

PrimaryNumber of Participants Experiencing a Serious Adverse Event (SAE).
Time frame:
The safety assessment period is approximately 9 weeks.
Reported as:
Count of participants · Participants
Number of Participants Experiencing a Serious Adverse Event (SAE).
ParticipantsTopical NS2 1% Dermatologic CreamTopical Vehicle Dermatologic
Number of Participants Experiencing a Serious Adverse Event (SAE).00
PrimaryNumber of Participants Experiencing an Adverse Event Leading to Discontinuation.
Time frame:
The safety assessment period is approximately 9 weeks.
Reported as:
Count of participants · Participants
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
ParticipantsTopical NS2 1% Dermatologic CreamTopical Vehicle Dermatologic
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.00

Adverse events

Collected over Adverse event data was captured for approximately 9 weeks for each subject during clinical trial participation.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Topical NS2 1% Dermatologic Cream0/6 (0%)0/6 (0%)5/6 (83.3%)
Topical Vehicle Dermatologic0/6 (0%)0/6 (0%)3/6 (50%)
Most frequent other events
Showing 10 of 18
Most frequent other events
EventTopical NS2 1% Dermatologic CreamTopical Vehicle Dermatologic
PruritusSkin and subcutaneous tissue disorders2/60/6
BlisterSkin and subcutaneous tissue disorders0/61/6
RashSkin and subcutaneous tissue disorders0/61/6
Skin ulcerSkin and subcutaneous tissue disorders0/61/6
NasopharyngitisInfections and infestations0/61/6
Tooth abscessInfections and infestations1/60/6
Upper respiratory tract infectionInfections and infestations1/60/6
ArthralgiaMusculoskeletal and connective tissue disorders1/61/6
Joint swellingMusculoskeletal and connective tissue disorders0/61/6
MyalgiaMusculoskeletal and connective tissue disorders1/60/6

Baseline characteristics

Age, Continuous
Age, Continuous(years)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Mean14.2 ± 5.0817.3 ± 5.0115.8 ± 5.08
Sex: Female, Male
Sex: Female, Male(Participants)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Female336
Male336
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Hispanic or Latino101
Not Hispanic or Latino5611
Unknown or Not Reported000
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Asian000
Black or African American213
Native Hawaiian or Pacific Islander000
White358
Other101
Region of Enrollment
Region of Enrollment(Participants)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
United States6612
Height
Height(centimeters)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Mean150.0 ± 19.71157.2 ± 9.14153.6 ± 15.13
Weight
Weight(kilograms)Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Mean41.4 ± 13.0759.8 ± 14.3650.6 ± 16.24
Body Surface Area
Body Surface Area(Mosteller Method (m2))Topical NS2 1% Dermatologic CreamTopical Vehicle DermatologicTotal
Mean1.54 ± 0.5271.95 ± 0.8901.77 ± 0.743
07

Study locations

3 sites
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198, United States
  • Penn State Hershey Medical Center - Department of Dermatology
    Hershey, Pennsylvania 17033, United States
  • Pediatric and General Dermatology
    Fairfax, Virginia 22031, United States
08

Registry details

Key details

Study ID
NCT02402309
Lead sponsor
Aldeyra Therapeutics, Inc.
Responsible party
Sponsor
First posted
Mar 30, 2015
Start date
Mar 2015
Primary completion
May 2016
Completion
Jun 2016
Results posted
Dec 13, 2022
Last update
Dec 13, 2022

Study contacts

William B. Rizzo, MD
principal investigator · University of Nebraska

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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