Long-Term, Observational, Global Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies (C5ITs)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 52
- 主要终点
- Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score
研究概览
简要总结
Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).
详细描述
At the time of enrollment in the Registry, participant records will be queried for retrospective information about the participants' medical history and gMG disease treatment history. Following enrollment, prospective data collection will be performed using data obtained as part of the routine clinical care and through patient-reported outcome methods in use. Data will be collected using an electronic data capture system. The duration of data collection for the Registry will be up to 5 years from the day of enrollment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Participants with a diagnosis of gMG who are treated with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.
- •Capable of giving signed informed consent, which includes compliance with the protocol requirements and restrictions.
- •Participants must have myasthenia gravis historical data, such as MG-ADL and MGFA class, available to be enrolled in the Registry.
排除标准
- •1. Participants currently enrolled in an Alexion-sponsored interventional clinical study for treatment of gMG cannot be enrolled in the Alexion gMG Registry while enrolled/participating in the clinical study for gMG therapy.
结局指标
主要结局
Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score
时间窗: Approximately 5 years from enrollment
The MG-ADL is an eight-item patient-reported outcome measure assessing MG symptoms and functional activities related to activities of daily living.48 Each of the items is scored from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24, where higher scores indicate greater severity of symptoms.
次要结局
未报告次要终点