Sjögren-Larsson SyndromePhase 2NCT02402309
A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
A Phase 2 study testing Active topical NS2 1% dermatologic cream in people with Sjögren-Larsson Syndrome, run by Aldeyra Therapeutics, Inc. at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Sjögren-Larsson Syndrome
- What is being tested?
- Active topical NS2 1% dermatologic cream
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 3 sitesUnited States
- Study dates
- Mar 2015to Jun 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically-confirmed diagnosis of SLS
- Active ichthyosis on the lower extremities that is determined to be at least moderate severity
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Pediatric and General Dermatology
Fairfax, Virginia, 22031
Penn State Hershey Medical Center - Department of Dermatology
Hershey, Pennsylvania, 17033
University of Nebraska Medical Center
Omaha, Nebraska, 68198
About this study
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)
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.
- Conditions
- Sjögren-Larsson Syndrome
- Being tested
- Active topical NS2 1% dermatologic cream, Vehicle placebo 0.0% NS2 dermatologic cream
- 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
- Medical subject headings
- Sjogren-Larsson Syndrome, Ichthyosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Active topical NS2 1% dermatologic creamDrug
Vehicle placebo 0.0% NS2 dermatologic creamDrug
| Study group | Role | What they receive |
|---|---|---|
| Active topical NS2 1% dermatologic cream | Experimental | NS2 1% topical cream for dermal application Receives: Drug: Active topical NS2 1% dermatologic cream |
| Topical vehicle dermatologic | Placebo comparator | Vehicle placebo for dermal application Receives: Drug: Vehicle placebo 0.0% NS2 dermatologic cream |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing a Serious Adverse Event (SAE).
Measured: The safety assessment period is approximately 9 weeks.
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
Measured: The safety assessment period is approximately 9 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2015 (estimated)
- Study startMar 2015
- Primary completionMay 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sjögren-Larsson SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aldeyra Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- William B. Rizzo, MD, Principal investigator, University of Nebraska
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02402309
- Sponsor's protocol ID
- NS-003
- Status last verified
- Nov 2022
- First submitted
- 17 Mar 2015
- Last updated
- 13 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02402309
- Acronym / sponsor ID
- NS-003
- Conditions
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Aldeyra Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2015
- Primary completion
- May 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 30 Mar 2015 (estimated)
- Results
- Posted 13 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active topical NS2 1% dermatologic cream | Experimental | Drug: Active topical NS2 1% dermatologic creamNS2 1% topical cream for dermal application |
| Topical vehicle dermatologic | Placebo comparator | Drug: Vehicle placebo 0.0% NS2 dermatologic creamVehicle placebo for dermal application |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Genetically-confirmed diagnosis of SLS
- Active ichthyosis on the lower extremities that is determined to be at least moderate severity
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Active topical NS2 1% dermatologic cream | Drug | Active topical NS2 1% dermatologic cream |
| Vehicle placebo 0.0% NS2 dermatologic cream | Drug | Topical vehicle dermatologic |
Primary outcome
Number of Participants Experiencing a Serious Adverse Event (SAE).
Time frame: The safety assessment period is approximately 9 weeks.
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
Time frame: The safety assessment period is approximately 9 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Sjögren-Larsson Syndrome
RecruitingObservational study
The Myelin Disorders Biorepository Project
Leukodystrophy, White Matter Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
William B. Rizzo, MD principal investigator
University of Nebraska
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Active topical NS2 1% dermatologic cream in people with Sjögren-Larsson Syndrome, run by Aldeyra Therapeutics, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Experiencing a Serious Adverse Event (SAE). (time frame: The safety assessment period is approximately 9 weeks.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Dec 2022.