Neuromyelitis OpticaPhase 1/2NCT02865018
Neuromyelitis Optica (NMO) & Cetirizine
A Phase 1/2 study testing cetirizine in people with Neuromyelitis Optica, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
Trial summary
Neuromyelitis optica (NMO) is an autoimmune disease that affects the central nervous system. Patients have relapses (also known as attacks) which are often quite severe and leave them with significant disability. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Neuromyelitis Optica
- What is being tested?
- cetirizine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2014to Feb 2016
- Phase
- Phase 1/2
- 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 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years to 85
- Meet criteria for the diagnosis of neuromyelitis optica as outlined by Wingerchuk et al in 2006. Alternatively patients may be included if they have had an episode of myelitis or optic neuritis in combination with a positive NMO IgG antibody, as positive antibody with a first episode is highly associated with future relapse.
- Disease duration of at least 6 months
- Stable, without any NMO relapses, for the 3 months prior to the baseline assessment visit
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Who cannot join
- Current therapy with daily cetirizine or another daily antihistamine for any indication
- Known hypersensitivity to cetirizine, hydroxyzine, or any component of the formulation
- Change in NMO disease-modifying therapy in the 3 months prior to baseline assessment
- Pregnancy or planning pregnancy during the study period
- Severe renal or hepatic impairment
- Inability to complete the study protocol for any reason
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: An Open Label, add-on Trial of Cetirizine for Patients With Neuromyelitis Optica
Neuromyelitis optica (NMO) is an autoimmune disease that affects the central nervous system. Patients have relapses (also known as attacks) which are often quite severe and leave them with significant disability. Without treatment, within 5 years 50% of NMO patients are blind in one or both eyes or require walking assistance (cane, walker or wheelchair).
NMO has only been relatively recently described and is fairly rare. Most NMO patients' immune systems produce abnormal antibodies against aquaporin-4 (AQP4), which is found in certain cells in the central nervous system. When these AQP4 antibodies bind to AQP4, they trigger a cascade of events involving the immune system which eventually leads to damage to the nervous system. This ultimately leads to disability, some of which is permanent.
Read more
Until now, treatments for NMO have been mostly focused on decreasing production of this AQP4 antibody. However, recent experiments in animal models of NMO have shown the importance of what happens inside the central nervous system after the antibody binds to the nervous system cell. Specifically, researchers have noted the importance of a specific cell type, eosinophils, in causing damage in NMO lesions. In a recent study, researchers showed they could prevent damage from NMO by blocking eosinophils using cetirizine, which is a popular over-the-counter allergy medicine.
Cetirizine is already known to be safe and well-tolerated in the general population. In this study, the researchers plan to add cetirizine on to patients' current NMO treatment. The researchers aim to show that it is safe, well-tolerated, and that with cetirizine, NMO patients have less relapses and therefore less disability over the course of the year following initiation of treatment. The researchers also plan to study how cetirizine changes the immunological profile in NMO patients by examining blood and cerebrospinal fluid.
Read the detailed description
The researchers hypothesize that cetirizine, an allergy medication that acts as an eosinophil-stabilizer, will decrease the relapse rate when added to current standard therapy in patients with neuromyelitis optica.
Medication compliance will be assessed by the research coordinator at each visit through discussion with the patient and pill counting.
- Conditions
- Neuromyelitis Optica
- Being tested
- cetirizine
- Keywords
- Neuromyelitis Optica, Cetirizine
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cetirizineDrug
Also called Zyrtec
| Study group | Role | What they receive |
|---|---|---|
| cetirizine | Experimental | 10mg oral each day Receives: Drug: cetirizine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Relapse Rate Before Cetirizine
Relapses defined as "patient-reported symptoms or objectively observed signs typical of an acute inflammatory demyelinating event in the CNS, with duration of at least 24 hours, in the absence of fever or infection." The on study ARR was calculated as the number of relapses during the study divided by the length of time in the study.
Measured: Baseline and 1 year
Secondary outcomes (3)
Epworth Sleepiness Scale
Sedation as measured by Epworth Sleepiness Scale. The test is a list of eight situations in which you rate your tendency to become sleepy on a scale of 0, no chance of dozing, to 3, high chance of dozing. Total score 0 to 24 from unlikelihood of abnormally sleep to excessively sleepy.
Measured: Baseline and 1 year
Expanded Disability Status Scale (EDSS)
Disability as measured by Expanded Disability Status Scale (EDSS). The EDSS provides a total score on a scale from 0 to 10, from normal function to lessening function with higher score, 10, being death due to MS.
Measured: Baseline and 1 year
Eotaxin Plasma Levels
Eotaxin - an eosinophil-specific chemoattractant in the blood. Immunological measures related to eosinophil activity. Eotaxin plasma levels.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2016 (estimated)
- Study startApr 2014
- Primary completionFeb 2016
- Study completionFeb 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 Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Icahn School of Medicine at Mount SinaiThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Sponsor type
- University, hospital or other
- Collaborators
- Guthy Jackson Charitable Foundation
- Responsible party
- Principal investigator, Ilana Katz Sand, Icahn School of Medicine at Mount Sinai
- Investigators
- Ilana Katz Sand, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02865018
- Sponsor's protocol ID
- 13-1513
- Status last verified
- May 2023
- First submitted
- 9 Aug 2016
- Last updated
- 7 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, add-on Trial of Cetirizine for Patients With Neuromyelitis Optica
- ClinicalTrials.gov ID
- NCT02865018
- Acronym / sponsor ID
- 13-1513
- Conditions
- Neuromyelitis Optica
- Keywords
- Neuromyelitis Optica, Cetirizine
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Icahn School of Medicine at Mount SinaiUniversity, hospital or other
- Collaborators
- Guthy Jackson Charitable Foundation
- Study start
- Apr 2014
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 12 Aug 2016 (estimated)
- Results
- Posted 7 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neuromyelitis optica (NMO) is an autoimmune disease that affects the central nervous system. Patients have relapses (also known as attacks) which are often quite severe and leave them with significant disability. Without treatment, within 5 years 50% of NMO patients are blind in one or both eyes or require walking assistance (cane, walker or wheelchair).
NMO has only been relatively recently described and is fairly rare. Most NMO patients' immune systems produce abnormal antibodies against aquaporin-4 (AQP4), which is found in certain cells in the central nervous system. When these AQP4 antibodies bind to AQP4, they trigger a cascade of events involving the immune system which eventually leads to damage to the nervous system. This ultimately leads to disability, some of which is permanent.
Until now, treatments for NMO have been mostly focused on decreasing production of this AQP4 antibody. However, recent experiments in animal models of NMO have shown the importance of what happens inside the central nervous system after the antibody binds to the nervous system cell. Specifically, researchers have noted the importance of a specific cell type, eosinophils, in causing damage in NMO lesions. In a recent study, researchers showed they could prevent damage from NMO by blocking eosinophils using cetirizine, which is a popular over-the-counter allergy medicine.
Cetirizine is already known to be safe and well-tolerated in the general population. In this study, the researchers plan to add cetirizine on to patients' current NMO treatment. The researchers aim to show that it is safe, well-tolerated, and that with cetirizine, NMO patients have less relapses and therefore less disability over the course of the year following initiation of treatment. The researchers also plan to study how cetirizine changes the immunological profile in NMO patients by examining blood and cerebrospinal fluid.
Detailed description
The researchers hypothesize that cetirizine, an allergy medication that acts as an eosinophil-stabilizer, will decrease the relapse rate when added to current standard therapy in patients with neuromyelitis optica.
Medication compliance will be assessed by the research coordinator at each visit through discussion with the patient and pill counting.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| cetirizine | Experimental | Drug: cetirizine10mg oral each day |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 18 years to 85
- Meet criteria for the diagnosis of neuromyelitis optica as outlined by Wingerchuk et al in 2006. Alternatively patients may be included if they have had an episode of myelitis or optic neuritis in combination with a positive NMO IgG antibody, as positive antibody with a first episode is highly associated with future relapse.
- Disease duration of at least 6 months
- Stable, without any NMO relapses, for the 3 months prior to the baseline assessment visit
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Key exclusion criteria (6 listed)
- Current therapy with daily cetirizine or another daily antihistamine for any indication
- Known hypersensitivity to cetirizine, hydroxyzine, or any component of the formulation
- Change in NMO disease-modifying therapy in the 3 months prior to baseline assessment
- Pregnancy or planning pregnancy during the study period
- Severe renal or hepatic impairment
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 |
|---|---|---|
| cetirizineAlso: Zyrtec | Drug | cetirizine |
Primary outcome
Annualized Relapse Rate Before Cetirizine
Time frame: Baseline and 1 year
Relapses defined as "patient-reported symptoms or objectively observed signs typical of an acute inflammatory demyelinating event in the CNS, with duration of at least 24 hours, in the absence of fever or infection." The on study ARR was calculated as the number of relapses during the study divided by the length of time in the study.
3 secondary outcomes
Epworth Sleepiness Scale
Time frame: Baseline and 1 year
Expanded Disability Status Scale (EDSS)
Time frame: Baseline and 1 year
Eotaxin Plasma Levels
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Ilana Katz Sand, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing cetirizine in people with Neuromyelitis Optica, run by Icahn School of Medicine at Mount Sinai at 1 site 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 5 inclusion and 6 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 7 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 7 Jun 2023). 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?
Annualized Relapse Rate Before Cetirizine (time frame: Baseline and 1 year). 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 7 Jun 2023; AzTrials last refreshed its copy on 6 Nov 2026.