A Phase III, Open-label, Extension Trial of ECU-NMO-301 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
- 已完成
- 入组人数
- 119
- 试验地点
- 124
- 主要终点
- Number of Participants With An On-trial Relapse as Determined by The Treating Physician
研究概览
简要总结
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.
详细描述
This study is an open label extension study to confirm the long term safety and efficacy of eculizumab in subjects with relapsing NMO who have completed the initial double-blind, randomized, placebo-controlled trial ECU-NMO-301.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient completed the ECU-NMO-301 trial
- •Patient has given written informed consent
排除标准
- •Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to trial drug
- •Female patients who are pregnant, breastfeeding, or intend to conceive during the course of the trial
结局指标
主要结局
Number of Participants With An On-trial Relapse as Determined by The Treating Physician
时间窗: Baseline up to end of study (up to 6.5 years)
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.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
时间窗: Baseline up to end of study (up to 6.5 years)
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
时间窗: Baseline up to end of study (up to 6.5 years)
The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent.
On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
时间窗: Baseline up to end of study (up to 6.5 years)
The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.
次要结局
- Change From Baseline in Modified Rankin Scale (mRS) Score(Baseline, Weeks 52, 104 and 156)
- Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function(Baseline, Weeks 52, 104 and 156)
- Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score(Baseline, Weeks 52, 104 and 156)
- Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function(Baseline, Weeks 52, 104 and 156)
- Change From Baseline in Expanded Disability Status Scale (EDSS) Score(Baseline, Weeks 52, 104 and 156)