A Multicenter Non Interventional Single Arm Retrospective-prospective Observational Study in Therapeutic Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis (gMG) in Real Clinical Practice in Russia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- AstraZeneca
- 入组人数
- 450
- 试验地点
- 8
- 主要终点
- Disease duration (days)
研究概览
简要总结
This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in routine clinical practice in Russia.
详细描述
This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in routine clinical practice in Russia.
The primary objective is to characterize the demographic and clinical profile of adult AChR-Ab-positive gMG patients. Key secondary objectives include assessing disease severity over time (MG-ADL, QMG, MGFA class), evaluating rates, duration, and reasons for all-cause and gMG-related hospitalizations, describing diagnostic pathways and treatment strategies (including first-line regimens and use of therapies such as anticholinesterase agents, corticosteroids, immunosuppressants, IVIG, plasmapheresis/plasma filtration, thymectomy, rituximab, and complement C5 inhibitors), exploring patterns and outcomes of myasthenic crises, and documenting meningococcal vaccination and any prophylactic antibiotic use prior to initiation of C5 inhibitor therapy.
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. Data are collected from existing paper/electronic medical records (secondary data collection) and recorded into an eCRF at seven timepoints: baseline (with retrospective abstraction, including medical history from diagnosis) and prospective follow-up every 6 months up to Month 36 (±1 month windows). No study-mandated interventions, tests, or visit schedules are imposed; all care follows routine practice.
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) diagnosed with generalized MG positive for acetylcholine receptor (AChR) antibodies.
- •Provision of signed and dated written informed consent.
排除标准
- •Participants currently enrolled in clinical studies for treatment of gMG.
- •Ocular MG only.
结局指标
主要结局
Disease duration (days)
时间窗: Baseline (Month 0)
Time between the date of documented gMG diagnosis and the date of informed consent/enrollment, summarized as median and IQR (and mean, SD, min, max). Calculated in months/years based on dates abstracted from medical records to the eCRF.
Age at baseline (years)
时间窗: Baseline (Month 0; at informed consent and enrollment)
Patient age in full years at study enrollment (ICF signing). Summarized as a quantitative variable and as categorical distribution by ≤50 years vs \>50 years.
Age at gMG diagnosis (years)
时间窗: Baseline (retrospective abstraction at Month 0)
Patient age in full years at the date of generalized myasthenia gravis (gMG) diagnosis. Summarized as a quantitative variable and as categorical distribution by ≤50 years vs \>50 years.
Sex and ethnicity distribution
时间窗: Baseline (Month 0)
Proportion of patients by sex (male, female) and ethnicity (White, Asian, Black, other) recorded at enrollment.
Body Mass Index (BMI)
时间窗: Baseline (Month 0; last available measurements closest to ICF signing)
BMI in kg/m\^2 calculated as weight (kg)/height (m)\^2 using the most recent values closest to enrollment, summarized as mean, SD, median, IQR, minimum, maximum. Height and weight are abstracted from medical records.
Clinical symptom profile of gMG
时间窗: Baseline (Month 0)
Proportion of patients presenting each predefined gMG symptom category at enrollment (ocular, bulbar, respiratory, chewing/facial, limb/trunk/neck weakness, autonomic dysfunction subdomains); summarized as counts and percentages.
Comorbidity profile
时间窗: Baseline (Month 0)
Proportion of patients with specified comorbidities in history or present at enrollment: autoimmune thyroiditis/hyperthyroidism, systemic lupus erythematosus, rheumatoid arthritis, autoimmune encephalitis, arterial hypertension, hyperlipidemia.
Number and proportion of patients with gMG-related hospitalizations
时间窗: Retrospective period from gMG diagnosis to Baseline (Month 0)
Patients with ≥1 hospitalization attributed to gMG (investigator judgment) from date of gMG diagnosis to ICF signing.
Duration of gMG-related hospitalizations (days)
时间窗: Retrospective period from gMG diagnosis to Baseline (Month 0)
Sum of days of gMG-related hospitalizations per patient from gMG diagnosis to ICF signing.
次要结局
- MG-ADL total score and change from baseline(Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month))
- QMG total score and change from baseline(Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month))
- Distribution of MGFA clinical classification over time(Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month))
- Number and proportion of patients with all-cause hospitalization(From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month))
- Duration of all-cause hospitalizations (days per participant)(From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month))
- Number and proportion of patients with gMG-related hospitalization(From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month))
- Duration of gMG-related hospitalizations (days per participant)(From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month))
- Time from MG symptom onset to gMG diagnosis (months)(Retrospective period prior to Baseline)
- Prior misdiagnoses before gMG diagnosis (number and proportion by category)(Retrospective period prior to Baseline)
- Diagnostic examinations for gMG confirmation and available results(Retrospective period prior to Baseline)
- Diagnostic test usage and results(Retrospective period prior to Baseline)
- Drug therapies received (proportion by class)(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Duration of drug therapies (days per participant)(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Mean daily corticosteroid dose (prednisolone equivalent)(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Plasmapheresis/plasma exchange/plasmafiltration use (proportion)(12 months pre-Baseline and Baseline to Month 36. (±1 month))
- Duration of plasmapheresis/plasma exchange/plasmafiltration (days)(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Thymectomy performed(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Invasive lung ventilation required(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- First-line treatment regimen at gMG diagnosis(At gMG diagnosis (retrospective abstraction at Baseline))
- Myasthenic crisis in prior 12 months(12 months prior to Baseline (Month 0))
- Myasthenic crisis during follow-up(From Baseline to Month 36 (±1 month))
- Crisis precipitants-physical stressors(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Crisis precipitants-medications(12 months pre-Baseline and Baseline to Month 36 (±1 month))
- Crisis outcomes by MGFA class change(Months 6, 12, 18, 24, 30, 36 (±1 month))
- Initiation of complement C5 inhibitor therapy (C5IT)(From gMG diagnosis to Month 36 (±1 month))
- Meningococcal vaccination prior to C5IT initiation(From gMG diagnosis to C5IT start (vaccination events abstracted during follow-up))
- Prophylactic antibiotic use prior to C5IT initiation(From gMG diagnosis to C5IT start (antibiotic events abstracted during follow-up))