Sjogren-Larsson Syndrome
Conditions
Keywords
ADX-102, Sjögren-Larsson Syndrome
Brief summary
A Phase 3 Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Trial to Evaluate the Safety and Efficacy of ADX-102 1% Topical Dermal Cream in Subjects with Sjögren-Larsson Syndrome (SLS).
Interventions
DRUGADX-102 1% Topical Dermal Cream (reproxalap)
ADX-102 1% Topical Dermal Cream administered approximately every 24 hours for 6 months.
DRUGVehicle of ADX-102 Topical Dermal Cream
Vehicle of ADX-102 Topical Dermal Cream administered approximately every 24 hours for 6 months.
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
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject is aged 3 years or older * Subject has a genetically-confirmed diagnosis of SLS. * Subject has active ichthyosis that is Grade 2 or higher on the Visual Index Ichthyosis Severity (VIIS) score. * Females of child-bearing potential: Negative pregnancy test at Screening and Baseline Visits.
Exclusion criteria
* Subject has evidence of a serious active infection. * Systemic or topical retinoids or other topical medications, not including emollients, within the past 30 days Baseline Visit 1. * Subject has received an investigational systemic or topically administered drug within the past 30 days prior to Baseline Visit 1. * Subject is currently receiving immunosuppressive therapy, including intermittent or low-dose systemic corticosteroids. * Subject has a known allergic reaction to any ingredients of study drug formulation. * Subject has any clinically significant laboratory test abnormalities or a history of any other condition that, in the opinion of the Investigator, could compromise the subject's ability to comply with the protocol or that could compromise the subject's safety or the interpretation of the study results.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Visual Index Ichthyosis Severity (VIIS) Scaling Score as Assessed by the Investigator in ADX-102 Treated Patients | Efficacy assessment period (Week 1 through Week 24), where Baseline is Day 1. | Change from baseline using the Visual Index Ichthyosis Severity (VIIS) Scaling Score (0 = least, 4 = most), where a higher value represents a worse outcome, in ADX-102 treated patients |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| ADX-102 1% Topical Dermal Cream ADX-102 1% Topical Dermal Cream: ADX-102 1% Topical Dermal Cream administered approximately every 24 hours for 6 months in Part 1 | 7 |
| Vehicle Topical Dermal Cream Vehicle Topical Dermal Cream: Vehicle of ADX-102 Topical Dermal Cream administered approximately every 24 hours for 6 months in Part 1 | 4 |
| Total | 11 |
Baseline characteristics
| Characteristic | Total | Vehicle Topical Dermal Cream | ADX-102 1% Topical Dermal Cream |
|---|---|---|---|
| Age, Continuous | 15.0 years STANDARD_DEVIATION 8.21 | 9.5 years STANDARD_DEVIATION 6.35 | 18.1 years STANDARD_DEVIATION 7.78 |
| Body Mass Index | 19.1 kg/m^2 STANDARD_DEVIATION 3.57 | 16.8 kg/m^2 STANDARD_DEVIATION 2.89 | 20.5 kg/m^2 STANDARD_DEVIATION 3.36 |
| Body Surface Area | 1.3 m^2 STANDARD_DEVIATION 0.43 | 1.0 m^2 STANDARD_DEVIATION 0.42 | 1.4 m^2 STANDARD_DEVIATION 0.39 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 9 Participants | 3 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Height | 141.39 centimeters STANDARD_DEVIATION 27.85 | 125.68 centimeters STANDARD_DEVIATION 29.287 | 150.38 centimeters STANDARD_DEVIATION 24.583 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 3 Participants | 1 Participants | 2 Participants |
| Race/Ethnicity, Customized Native Hawaiian or other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized White | 8 Participants | 3 Participants | 5 Participants |
| Region of Enrollment United States | 11 Participants | 4 Participants | 7 Participants |
| Sex: Female, Male Female | 6 Participants | 3 Participants | 3 Participants |
| Sex: Female, Male Male | 5 Participants | 1 Participants | 4 Participants |
| Weight | 41.67 kilograms (kg) STANDARD_DEVIATION 20.367 | 29.18 kilograms (kg) STANDARD_DEVIATION 18.864 | 48.81 kilograms (kg) STANDARD_DEVIATION 18.708 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 4 |
| other Total, other adverse events | 6 / 7 | 2 / 4 |
| serious Total, serious adverse events | 0 / 7 | 0 / 4 |
Outcome results
Primary
Visual Index Ichthyosis Severity (VIIS) Scaling Score as Assessed by the Investigator in ADX-102 Treated Patients
Change from baseline using the Visual Index Ichthyosis Severity (VIIS) Scaling Score (0 = least, 4 = most), where a higher value represents a worse outcome, in ADX-102 treated patients
Time frame: Efficacy assessment period (Week 1 through Week 24), where Baseline is Day 1.
Population: modified intent to treat population
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| ADX-102 1% Topical Dermal Cream | Visual Index Ichthyosis Severity (VIIS) Scaling Score as Assessed by the Investigator in ADX-102 Treated Patients | -0.7 units on a scale | Standard Error 0.22 |