Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients With Anti-AChR Myasthenia Gravis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 55
- 试验地点
- 1
- 主要终点
- MG-ADL scores
研究概览
简要总结
This study called "Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients with Anti-AChR Myasthenia Gravis", is investigating the effects of a new treatment called Zilucoplan (generic name: ZILBRYSQ) on patients in France with a condition known as myasthenia gravis. Myasthenia gravis is an autoimmune disease that causes muscle weakness that worsens throughout the day with activity and impacts the ability to work and perform daily activities. The study aims to see if Zilucoplan can improve muscle strength and quality of life over three months, while also assessing its tolerance and safety by monitoring side effects. Researchers hope this treatment will offer significant benefits for people with this challenging condition.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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
- •- Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan
排除标准
- 未提供
研究组 & 干预措施
Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan
干预措施: Zilbrysq (Drug)
结局指标
主要结局
MG-ADL scores
时间窗: At the inclusion, at 1 month, at 3 months and at 6 months
The MG-ADL is an 8-item patient-reported scale that measures myasthenia gravis symptoms and functional status. It assesses the impact of Myasthenia Gravis on the daily functions of the patients
次要结局
- Garches scale scores(At the inclusion, at 1 month, at 3 months and at 6 months)
- Evaluation of tolerance (M6) through the description of type of Adverse events(At 6 months)
- Evaluation of tolerance (M6) through the description of the frequency of Adverse event(At 6 months)
- Adverse events possible association with the treatment (causality)(At 6 months)
- Quality of life evaluation (MG-QoL15r)(At the inclusion, at 1 month, at 3 months and at 6 months)