Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia

A Multicenter Non-interventional Single-arm Retrospective-prospective Observational Study in Therapeutic Approaches on AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) in Real Clinical Practice in Russia

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.

Study Overview

• Status: Suspended
• Conditions: Rare Diseases; Neuromyelitis Optica Spectrum Disorder (NMOSD)

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

Study Locations

• Russia
  • Novosibirsk, Russia
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

Inclusion Criteria:

1. Adults (≥18 years) with a confirmed diagnosis of AQP4-IgG positive NMOSD following the 2015 IPND criteria;
2. Provision of signed and dated written informed consent.

Exclusion Criteria:

1.Participants currently enrolled in clinical studies for the treatment of NMOSD.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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.	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

Secondary Outcome Measures

Outcome Measure	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	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)

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): December 23, 2025
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: November 18, 2025
• First Submitted That Met QC Criteria: November 18, 2025
• First Posted (Actual): November 25, 2025

Study Record Updates

• Last Update Posted (Actual): February 19, 2026
• Last Update Submitted That Met QC Criteria: February 17, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Pathological Conditions, Signs and Symptoms; Rare Diseases; Neuromyelitis Optica
• Other Study ID Numbers: D9282R00001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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
• IPD Sharing Time Frame: 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.
• IPD Sharing Access Criteria: 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.