Sjögren-Larsson Syndrome
Conditions
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
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.
Interventions
DRUGActive topical NS2 1% dermatologic cream
DRUGVehicle placebo 0.0% NS2 dermatologic cream
Sponsors
Aldeyra Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
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
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Topical NS2 1% Dermatologic Cream NS2 1% topical cream for dermal application
Active topical NS2 1% dermatologic cream | 6 |
| Topical Vehicle Dermatologic Vehicle placebo for dermal application
Vehicle placebo 0.0% NS2 dermatologic cream | 6 |
| Total | 12 |
Baseline characteristics
| Characteristic | Topical NS2 1% Dermatologic Cream | Topical Vehicle Dermatologic | Total |
|---|---|---|---|
| Age, Continuous | 14.2 years STANDARD_DEVIATION 5.08 | 17.3 years STANDARD_DEVIATION 5.01 | 15.8 years STANDARD_DEVIATION 5.08 |
| Body Surface Area | 1.54 Mosteller Method (m2) STANDARD_DEVIATION 0.527 | 1.95 Mosteller Method (m2) STANDARD_DEVIATION 0.89 | 1.77 Mosteller Method (m2) STANDARD_DEVIATION 0.743 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants | 6 Participants | 11 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Height | 150.0 centimeters STANDARD_DEVIATION 19.71 | 157.2 centimeters STANDARD_DEVIATION 9.14 | 153.6 centimeters STANDARD_DEVIATION 15.13 |
| Race/Ethnicity, Customized Asian | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 2 Participants | 1 Participants | 3 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized White | 3 Participants | 5 Participants | 8 Participants |
| Region of Enrollment United States | 6 Participants | 6 Participants | 12 Participants |
| Sex: Female, Male Female | 3 Participants | 3 Participants | 6 Participants |
| Sex: Female, Male Male | 3 Participants | 3 Participants | 6 Participants |
| Weight | 41.4 kilograms STANDARD_DEVIATION 13.07 | 59.8 kilograms STANDARD_DEVIATION 14.36 | 50.6 kilograms STANDARD_DEVIATION 16.24 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 6 |
| other Total, other adverse events | 5 / 6 | 3 / 6 |
| serious Total, serious adverse events | 0 / 6 | 0 / 6 |
Outcome results
Primary
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
Time frame: The safety assessment period is approximately 9 weeks.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Topical NS2 1% Dermatologic Cream | Number of Participants Experiencing an Adverse Event Leading to Discontinuation. | 0 Participants |
| Topical Vehicle Dermatologic | Number of Participants Experiencing an Adverse Event Leading to Discontinuation. | 0 Participants |
Primary
Number of Participants Experiencing a Serious Adverse Event (SAE).
Time frame: The safety assessment period is approximately 9 weeks.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Topical NS2 1% Dermatologic Cream | Number of Participants Experiencing a Serious Adverse Event (SAE). | 0 Participants |
| Topical Vehicle Dermatologic | Number of Participants Experiencing a Serious Adverse Event (SAE). | 0 Participants |