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As a part of portfolio and planning reassessment for the current period, a decision has been made to prioritize, optimize and reallocate resources. As a result, enrollment and other study activities have been temporarily paused.
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This is a multi-centre, retrospective-prospective, single-arm, non-interventional (observational) cohort study with secondary data collection within real-world settings of participants with AQP4-IgG positive NMOSD.
This is a multicenter, retrospective-prospective, single-arm observational cohort study using secondary data collection from routine care medical records.
The primary objective is to describe baseline demographic and clinical characteristics, diagnostic algorithms, and treatment approaches. Secondary objectives are to describe Expanded Disability Status Scale (EDSS) levels and dynamics, collect the rate, duration, and reasons for hospitalizations, and evaluate physician-reported relapse profiles.
Approximately 100 adults will be enrolled consecutively across about 10 specialized sites. The study will sequentially include only those patients who have signed the informed consent form (ICF). Eligible patients will be enrolled consecutively at each site to minimize selection bias. Each participant will be followed for 36 months from informed consent (T0), with data collection every 6 months (T1-T6). The baseline period is defined as the time from NMOSD diagnosis until inclusion, with retrospective data abstraction; subsequent data are collected prospectively at routine visits. All data are entered into an electronic case report form (eCRF) from paper/electronic medical records. No study-specific interventions are performed; treatment is determined by usual care.
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| Measure | Description | Time Frame |
|---|---|---|
| Baseline demographics and clinical characteristics | Summary of participant demographics and clinical history at inclusion: age at inclusion and at NMOSD diagnosis, sex, ethnicity, BMI at inclusion, disease duration, clinical symptoms at inclusion, comorbidities. | At inclusion |
| Pre-inclusion relapse history | Proportion of patients with ≥1 and >1 physician-reported relapses and severe relapses (EDSS increase ≥2.0 points from baseline per event); annualized relapse rate (ARR) prior to inclusion. | From NMOSD diagnosis to inclusion (retrospective baseline) |
| Pre-inclusion NMOSD-related hospitalizations | Number of patients with NMOSD-related hospitalizations from the time of NMOSD diagnosis; median cumulative duration (days) of NMOSD-related hospitalizations prior to inclusion. | From NMOSD diagnosis to inclusion (retrospective baseline) |
| Time from first symptoms to NMOSD diagnosis | Median time (months) from patient-reported first NMOSD symptoms to confirmed NMOSD diagnosis according to 2015 IPND criteria. | From first NMOSD symptoms to date of diagnosis (retrospective baseline) |
| Prior misdiagnoses profile | Proportion of patients with any prior misdiagnosis and by type (e.g., MS, MOGAD, CNS infections, SLE only, Sjögren's only, Behçet's, neurosarcoidosis, CNS vascular disease, toxic/metabolic, neoplasms/paraneoplastic, congenital CNS, other). | From first NMOSD symptoms to confirmed NMOSD diagnosis (retrospective baseline) |
| AQP4-IgG testing method | Number and proportion of patients tested by cell-based assay versus ELISA for AQP4-IgG serostatus determination. |
| Measure | Description | Time Frame |
|---|---|---|
| EDSS mean at each time point | Expanded Disability Status Scale (EDSS) mean score across participants at each scheduled assessment; EDSS assessed per routine clinical practice. | Baseline (T0) and Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6) |
| EDSS mean change from baseline |
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Inclusion Criteria:
Exclusion Criteria:
1.Participants currently enrolled in clinical studies for the treatment of NMOSD.
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Participants of both sexes aged 18 years and older with diagnosis of AQP4-IgG positive NMOSD established according to 2015 IPND criteria will be enrolled in various clinical institutions in Russia that provide treatment for NMOSD patients.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Novosibirsk | Russia |
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared
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AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
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| ID | Term |
|---|---|
| D035583 | Rare Diseases |
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D009188 | Myelitis, Transverse |
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| At time of NMOSD diagnostic workup (retrospective baseline) |
| MRI brain T2-hyperintense lesion count change | Mean change from baseline in the number of T2-hyperintense brain lesions; presence of T1 contrast-enhancing lesions recorded as categorical variables. | Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
| Optic nerve MRI findings | Presence of contiguous lesions, bilateral neuritis, chiasmal extension, and optic nerve atrophy recorded as categorical variables per timepoint. | Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
| Spinal cord MRI lesion metrics | T2 lesion count; presence of longitudinally extensive lesions (≥3 segments), transverse lesions, and spinal cord atrophy extending ≥3 segments recorded as categorical variables per timepoint. | Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
| Relapse prevention therapy patterns | Number of patients receiving relapse prevention therapy by regimen: immunosuppressive drugs monotherapy; biologic monotherapy; biologic plus immunosuppressive combination; mean daily corticosteroid dose if low-dose steroids used (prednisolone-equivalent). | From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
| Acute relapse treatment modalities | Number of patients receiving high-dose corticosteroids, plasma exchange, or immunoadsorption for acute relapses; summarized per period. | From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
| Concomitant medications | Number of patients by class/type of concomitant medications for comorbid conditions recorded from diagnosis to inclusion and prospectively. | From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion |
Mean change in EDSS from baseline (T0) to each follow-up time point; change calculated as EDSS at time point minus baseline EDSS. |
| Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6) |
| All-cause hospitalizations | Number of patients with ≥1 hospitalization from any cause during follow-up; counts summarized overall and by hospitalization cause categories. | From inclusion (T0) through Month 36 (T6) |
| Median cumulative duration of hospitalizations | Median cumulative number of days spent hospitalized per patient during follow-up; calculated across all hospitalizations per participant. | From inclusion (T0) through Month 36 (T6) |
| NMOSD-related hospitalizations | Number of patients with ≥1 hospitalization due to NMOSD during follow-up; NMOSD-related as documented in medical records. | From inclusion (T0) through Month 36 (T6) |
| Median cumulative duration of NMOSD-related hospitalizations | Median cumulative number of days spent hospitalized per patient for NMOSD-related causes during follow-up. | From inclusion (T0) through Month 36 (T6) |
| Patients with physician-reported relapse(s) | Number of patients with ≥1 and >1 physician-reported NMOSD relapse during follow-up; relapses defined per protocol and documented by clinician assessment. | From inclusion (T0) through Month 36 (T6) |
| Patients with severe relapse(s) | Number of patients with ≥1 and >1 severe NMOSD relapse during follow-up; severe relapse defined as EDSS increase ≥2.0 points from baseline (for myelitis) or major OSIS exacerbation, per protocol. | From inclusion (T0) through Month 36 (T6) |
| Annualized relapse rate (ARR) | ARR calculated as total number of physician-reported relapses divided by person-years observed during follow-up for each patient, summarized at the cohort level. | From inclusion (T0) through Month 36 (T6) |
| Median time to first physician-reported relapse | Time from inclusion (T0) to first physician-reported NMOSD relapse; analyzed using Kaplan-Meier methods with censoring at Month 36 or withdrawal. | From inclusion (T0) to first relapse event, up to Month 36 (T6) |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |