Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis (gMG) Naive to Complement Inhibitors. Prospective, Multicenter, Non-interventional Study (MG-ARCADIA).
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- AstraZeneca
- 入组人数
- 50
- 试验地点
- 16
- 主要终点
- Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
研究概览
简要总结
The Polish multicentre observational (non-interventional) study aiming to collect data on the management and clinical outcomes of patients with gMG that received ravulizumab.
详细描述
The Polish, prospective, multicenter, observational, cohort study designed to collect longitudinal data on the management and clinical outcomes of patients with gMG naive to complement inhibitors who received ravulizumab in the scope of routine clinical practice within the frames of National Drug Program (NDP) for up to 42 months (~ 6 months baseline and ~36 months follow-up).
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
- •Adult (aged ≥18 years) patients with gMG, naive to complement inhibitors, receiving ravulizumab treatment in the frames of NDP in Poland.
- •Patients willing to participate in the study and signed Informed Consent Form (ICF).
- •Vaccination against N. meningitidis
排除标准
- •Those who plan to participate in gMG clinical trial on/after the date of first ravulizumab infusion through NDP.
- •Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
研究组 & 干预措施
Ravulizumab
Open-lable arm with gMG patients who received ravulizumab treatment in the frames of NDP in Poland.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
时间窗: at 6 month
次要结局
- Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score(at month 12, 24, 36)
- Change from baseline in MG-ADL subcomponent scores(at month 6, 12, 24, 36)
- Change from baseline in Myasthenia Gravis (MG) composite scale(at month 6, 12, 24, 36)
- Proportion of patients who achieved response(at month 6, 12, 24, 36)
- Change in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score(at month 6, 12, 24, 36)
- Change from baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP)(at month 6, 12, 24, 36)
- Change from baseline in Patient Global Impression of Change (PGI-C)(at month 6, 12, 24, 36)
- Change from baseline in Neuro-Qol Short Form v1.0 - Fatigue(at month 6, 12, 24, 36)
- Change from baseline in Patient Health Questionnaire - 9 (PHQ-9)(at month 6, 12, 24, 36)