A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 174
- 试验地点
- 77
- 主要终点
- Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
研究概览
简要总结
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 74 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
- •Positive serology for acetylcholine receptor (AChR) autoantibodies
- •MG-ADL Score of ≥ 6 at Screening and Baseline
- •QMG score ≥ 12 at Screening and Baseline
- •No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
- •No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
排除标准
- •Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
- •History of meningococcal disease
- •Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
研究组 & 干预措施
0.3 mg/kg zilucoplan (RA101495)
干预措施: zilucoplan (RA101495) (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
时间窗: From Baseline to End of Treatment (Week 12)
The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.
次要结局
- Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score(From Baseline to End of Treatment (Week 12))
- Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score(From Baseline to End of Treatment (Week 12))
- Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period(From Baseline to End of Treatment (Week 12))
- Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score(From Baseline to End of Treatment (Week 12))
- Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy(End of Treatment (Week 12))
- Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy(End of Treatment (Week 12))
- Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12(End of Treatment (Week 12))
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)(From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up]))