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| Name | Class |
|---|---|
| Guthy Jackson Charitable Foundation | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| cetirizine | Experimental | 10mg oral each day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| cetirizine | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline and 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline and 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ilana Katz Sand, MD | Icahn School of Medicine at Mount Sinai | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
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Twenty-four potential participants were referred by treating physicians at the Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai between April 2014 and February 2015. Sixteen were enrolled between Aprill 2014 and February 2016.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cetirizine | 10mg oral each day for 1 year |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cetirizine | 10mg oral each day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Mean | Standard Deviation | relapses per year | Baseline and 1 year |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cetirizine | 10mg oral each day for 1 year | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Unilateral eye pain with movement | Eye disorders |
Small sample size and lack of a control group.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ilana Katz Sand | Icahn School of Medicine at Mount Sinai, Corrine Goldsmith Dickinson Center for MS | 212-241-6854 | ilana.katzsand@mssm.edu |
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| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D017332 | Cetirizine |
| ID | Term |
|---|---|
| D006919 | Hydroxyzine |
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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| 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. |
| Baseline and 1 year |
| Eotaxin Plasma Levels | Eotaxin - an eosinophil-specific chemoattractant in the blood. Immunological measures related to eosinophil activity. Eotaxin plasma levels. | 6 months |
| years |
|
| Age, Customized | Median | Inter-Quartile Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Positive NMO antibody in serum | Count of Participants | Participants |
|
| Type of NMO Preventative Treatment at Enrollment | Count of Participants | Participants |
|
| Duration of Current Treatment | Median | Inter-Quartile Range | months |
|
| Number of Previous Preventative NMO treatment types | Count of Participants | Participants |
|
| Oral Prednisone use at Enrollment | Count of Participants | Participants |
|
| Total pre-study relapses | Relapses were 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." | Median | Inter-Quartile Range | relapses |
|
| Total pre-study relapses while on current preventative treatment | Median | Inter-Quartile Range | relapses |
|
| Annualized Relapse Rate | The pre-study annualized relapse rate (ARR) was calculated by taking the number of relapses each participant had experienced after the start of their current reported NMO preventative treatment, divided by the length of time (in years) they had been receiving this treatment. | Median | Inter-Quartile Range | relapses per treatment year |
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline and 1 year |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline and 1 year |
|
|
|
| Secondary | Eotaxin Plasma Levels | Eotaxin - an eosinophil-specific chemoattractant in the blood. Immunological measures related to eosinophil activity. Eotaxin plasma levels. | Posted | Median | Inter-Quartile Range | pg/mL | 6 months |
|
|
|
| 15 |
| 0 |
| 15 |
| 3 |
| 15 |
| Blurry vision with difficulty distinguishing colors | Eye disorders |
|
| Neuro episode | Nervous system disorders | re-emergence of old symptoms |
|
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| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |