A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Trial to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 143
- 试验地点
- 70
- 主要终点
- Participants With An Adjudicated On-trial Relapse
研究概览
简要总结
The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants ≥ 18 years old.
- •Diagnosis of NMO or NMOSD.
- •AQP4 antibody seropositive.
- •Historical relapse of at least 2 relapses in the last 12 months or 3 relapses in the last 24 months with at least 1 relapse in the 12 months prior to the screening.
- •Expanded Disability Status Scale score ≤
- •If a participant entered the study receiving immunosuppressive therapy (IST) for relapse prevention, the participant must have been on a stable maintenance dose of IST(s), as defined by the treating physician, prior to Screening and must have remained on that dose for the duration of the study, unless the participant experienced a relapse.
- •Female participants of childbearing potential were to have a negative pregnancy test (serum human chorionic gonadotropin). Participants were required to practice an effective, reliable, and medically approved contraceptive regimen during the study and for up to 5 months following discontinuation of treatment.
排除标准
- •Use of rituximab within 3 months prior to Screening.
- •Use of mitoxantrone within 3 months prior to Screening.
- •Use of intravenous immunoglobulin within 3 weeks prior to Screening.
研究组 & 干预措施
Eculizumab
Biological/Vaccine: Eculizumab; Induction Period: Participants received eculizumab (900 milligrams [mg]) via intravenous (IV) infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4). This was followed by the Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.
干预措施: Eculizumab (Drug)
Placebo
Placebo contains the same buffer components without the active ingredient. Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4). This was followed by the Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.
干预措施: Placebo (Drug)
结局指标
主要结局
Participants With An Adjudicated On-trial Relapse
时间窗: Baseline, Up To 211 Weeks (End of Study)
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 relapse adjudication committee.
次要结局
- Adjudicated On-trial Annualized Relapse Rate (ARR)(Baseline, Up To 211 Weeks (End of Study))
- Change From Baseline In EDSS At End Of Study(Baseline, Up To 211 Weeks (End of Study))
- Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study(Baseline, Up To 211 Weeks (End of Study))
- Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study(Baseline, Up To 211 Weeks (End of Study))
- Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study(Baseline, Up To 211 Weeks (End of Study))
- Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study(Baseline, Up To 211 Weeks (End of Study))