NCT06967480招募中不适用
MG-EVOLUTION. Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis: A Real-World Study With Biomarker Analysis
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 23
- 主要终点
- Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 50
研究概览
简要总结
The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.
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
- •Diagnosis of MG confirmed by:
- •History of a positive serologic test for anti-AChR antibodies, and
- •One of the following:
- •History of abnormal neuromuscular transmission test demonstrated by singlefibre electromyography or repetitive nerve stimulation
- •History of positive anticholinesterase test (e.g., edrophonium chloride test); or
- •Patient demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician
- •Disease duration from MG onset ≤ 3 years before T-4 (Enrolment);
- •MGFA class IIb to IV;
- •Patient eligible for Ravulizumab treatment based on AIFA reimbursement criteria
- •Vaccination cycle for Neisseria meningitidis completed at least two weeks before Ravulizumab initiation or antibiotics chemoprophylaxis according to the SmPC.
排除标准
- •Patient unable to understand and sign the informed consent
- •Hypersensitivity to the active substance or to any of the excipients of the study product
- •Patient for whom the study product is contraindicated according to SmPC
- •Previous treatment with C5 inhibitors
- •Last rituximab infusion performed < 6 months before T-4 (Enrolment)
- •Last infusion with FcRn blockers performed < 3 months before T-4 (Enrollment)
- •Pregnant or lactating or planning a pregnancy during the study
- •Patient who plan to relocate during the study
- •Patient who are unsure of following the visit schedule
- •Patient unable to complete questionnaires
- •Previous or current participation to other interventional studies
研究组 & 干预措施
Ravulizumab
Participants will receive Ravulizumab until Week 50 ± 14 days after baseline or until discontinuation.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 50
时间窗: Week 50
Change From Baseline in MG-ADL Total Score at Week 50
时间窗: Baseline, Week 50
次要结局
- MG-ADL Total Score at Weeks 2, 10, and 26(Weeks 2, 10, and 26)
- Change From Baseline in MG-ADL Total Score at Weeks 2, 10 and 26(Baseline, Weeks 2, 10 and 26)
- Percentage of Participants With >=2- Point Reduction From Baseline in MG-ADL Total Score at Weeks 2, 10, 26, and 50(Baseline, Weeks 2, 10, 26, and 50)
- Quantitative Myasthenia Gravis (QMG) Total Score at Weeks 2, 10, 26, and 50(Weeks 2, 10, 26, and 50)
- Change From Baseline in QMG Total Score at Weeks 2, 10, 26 and 50(Baseline, Weeks 2, 10, 26 and 50)
- Percentage of Participants With >=3- Point Reduction From Baseline in QMG Total Score at Weeks 2, 10, 26, and 50(Baseline, Weeks 2, 10, 26, and 50)
- Myasthenia Gravis Composite (MGC) Total Score at Weeks 2, 10, 26, and 50(Weeks 2, 10, 26, and 50)
- Change From Baseline in MGC Total Score at Weeks 2,10, 26 and 50(Baseline, Weeks 2, 10, 26 and 50)
- Percentage of Participants With >=3- Point Reduction From Baseline in MGC Total Score at Weeks 2, 10, 26 and 50(Baseline, Weeks 2, 10, 26, and 50)
- Myasthenia Gravis Quality of Life 15-Item Scale - Revised (MG-QoL15r) Total Score at Weeks 10, 26, and 50(Weeks 10, 26, and 50)
- Change From Baseline in MG-QoL15r Total Score at Weeks 10, 26 and 50(Baseline, Weeks 10, 26 and 50)
- Hospital Anxiety and Depression Scale (HADS) Total Score at Weeks 10, 26, and 50(Weeks 10, 26, and 50)
- Change From Baseline in HADS Total Score at Weeks 10, 26 and 50(Baseline, Weeks 10, 26 and 50)
- Insomnia Severity Index Scale (ISI) Total Score at Weeks 10, 26, and 50(Weeks 10, 26, and 50)
- Change From Baseline in ISI Total Score at Weeks 10, 26 and 50(Baseline, Weeks 10, 26 and 50)
- Pain Visual Analogue Scale (Pain VAS) Total Score at Weeks 2, 10, 26, and 50(Weeks 2, 10, 26, and 50)
- Change From Baseline in Pain VAS Total Score at Weeks 2, 10, 26 and 50(Baseline, Weeks 2, 10, 26 and 50)
- Biomarkers analyses (complement system proteins, interleukins and miRNA serum levels)(Baseline, Week 10, 26, and 50)
- Percentage of Participants With Mean Dose Reduction From Baseline of Corticosteroids and Immunosuppressants (ISTs) Treatment at Weeks 2, 10, 26, and 50(Baseline, Weeks 2, 10, 26, and 50)
- Percentage of Participants in Different ISTs Status at Baseline, and at Weeks 2, 10, 26, and 50(Baseline, Weeks 2, 10, 26, and 50)
- Number of Clinical Deterioration Events(Baseline, Weeks 2, 10, 26, and 50)
- Percentage of Participants With Minimal Symptom Expression at Weeks 2, 10, 26, and 50(Weeks 2, 10, 26, and 50)
- Institute For Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ) Score at Weeks 10, 26, and 50(Weeks 10, 26, and 50)
- Change From Baseline in iMTA iPCQ Score at Weeks 10, 26 and 50(Baseline, Weeks 10, 26 and 50)
研究者
研究点 (23)
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