A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 58
- 试验地点
- 1
- 主要终点
- Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
研究概览
简要总结
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- •At least 1 attack or relapse in the last 12 months prior to the Screening Period.
- •Expanded Disability Status Scale score ≤
- •Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
- •Body weight ≥40 kilograms.
- •Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
排除标准
- •History of Neisseria meningitidis infection.
- •Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
- •Previously or currently treated with a complement inhibitor.
- •Use of rituximab or mitoxantrone within 3 months prior to Screening.
- •Use of IV immunoglobulin within 3 weeks prior to Screening.
研究组 & 干预措施
Ravulizumab
During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed).
After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 3 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.
干预措施: Ravulizumab (Biological)
结局指标
主要结局
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period
时间窗: Baseline up to 2.25 years (end of the Primary Treatment Period)
An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the independent relapse adjudication committee.
次要结局
- Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period(Baseline up to 2.25 years (end of the Primary Treatment Period))
- Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period(Baseline up to 2.25 years (end of the Primary Treatment Period))
- Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period(Baseline, up to 2.25 years (end of the Primary Treatment Period))
- Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period(Baseline, up to 2.25 years (end of the Primary Treatment Period))
- Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period(Baseline up to 2.25 years (end of the Primary Treatment Period))
- Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period(Baseline up to 2.25 years (end of the Primary Treatment Period))
- Serum Ravulizumab Concentration(Predose and end of infusion (EOI) at Week 26)
- Change From Baseline in Serum Free C5 Concentration at Week 26(Baseline, Week 26 (Predose and EOI))
- Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period(Baseline, Weeks 26, 50, 82, and 106)