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
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- AstraZeneca
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Baseline demographics and clinical characteristics
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults (≥18 years) with a confirmed diagnosis of AQP4-IgG positive NMOSD following the 2015 IPND criteria;
- •Provision of signed and dated written informed consent.
排除标准
- •1.Participants currently enrolled in clinical studies for the treatment of NMOSD.
结局指标
主要结局
Baseline demographics and clinical characteristics
时间窗: At inclusion
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.
Time from first symptoms to NMOSD diagnosis
时间窗: From first NMOSD symptoms to date of diagnosis (retrospective baseline)
Median time (months) from patient-reported first NMOSD symptoms to confirmed NMOSD diagnosis according to 2015 IPND criteria.
Prior misdiagnoses profile
时间窗: From first NMOSD symptoms to confirmed NMOSD diagnosis (retrospective baseline)
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).
AQP4-IgG testing method
时间窗: At time of NMOSD diagnostic workup (retrospective baseline)
Number and proportion of patients tested by cell-based assay versus ELISA for AQP4-IgG serostatus determination.
MRI brain T2-hyperintense lesion count change
时间窗: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Mean change from baseline in the number of T2-hyperintense brain lesions; presence of T1 contrast-enhancing lesions recorded as categorical variables.
Optic nerve MRI findings
时间窗: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Presence of contiguous lesions, bilateral neuritis, chiasmal extension, and optic nerve atrophy recorded as categorical variables per timepoint.
Spinal cord MRI lesion metrics
时间窗: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
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.
Pre-inclusion relapse history
时间窗: From NMOSD diagnosis to inclusion (retrospective baseline)
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.
Pre-inclusion NMOSD-related hospitalizations
时间窗: From NMOSD diagnosis to inclusion (retrospective baseline)
Number of patients with NMOSD-related hospitalizations from the time of NMOSD diagnosis; median cumulative duration (days) of NMOSD-related hospitalizations prior to inclusion.
Relapse prevention therapy patterns
时间窗: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
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).
Acute relapse treatment modalities
时间窗: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Number of patients receiving high-dose corticosteroids, plasma exchange, or immunoadsorption for acute relapses; summarized per period.
Concomitant medications
时间窗: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Number of patients by class/type of concomitant medications for comorbid conditions recorded from diagnosis to inclusion and prospectively.
次要结局
- EDSS mean at each time point(Baseline (T0) and Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6))
- EDSS mean change from baseline(Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6))
- All-cause hospitalizations(From inclusion (T0) through Month 36 (T6))
- Median cumulative duration of hospitalizations(From inclusion (T0) through Month 36 (T6))
- NMOSD-related hospitalizations(From inclusion (T0) through Month 36 (T6))
- Median cumulative duration of NMOSD-related hospitalizations(From inclusion (T0) through Month 36 (T6))
- Patients with physician-reported relapse(s)(From inclusion (T0) through Month 36 (T6))
- Patients with severe relapse(s)(From inclusion (T0) through Month 36 (T6))
- Annualized relapse rate (ARR)(From inclusion (T0) through Month 36 (T6))
- Median time to first physician-reported relapse(From inclusion (T0) to first relapse event, up to Month 36 (T6))