An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Median Number of Neuromyelitis Optica (NMO) Attacks Per Year
研究概览
简要总结
The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.
详细描述
It has been shown in some scientific studies that the the antibody marker specific for neuromyelitis optica (NMO), known as NMO-Immunoglobulin G (IgG), causes inflammation in brain tissues by activating a substance called complement. Complement can greatly increase the immune attack in the optic nerves (causing optic neuritis (ON)), spinal cords (causing transverse myelitis (TM)) and brains of patients with NMO. Eculizumab has already been shown to be effective in a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). Attacks of PNH are also mediated through complement. Therefore, the investigators of this study are investigating whether by 'turning off' complement in NMO, further attacks of NMO can be prevented.
The primary (most important) objectives of this study are to determine:
Whether Eculizumab reduces relapse frequency in patients with relapsing NMO. The number of attacks during the one year treatment period will be compared to the number of attacks that occurred prior to initiation of eculizumab treatment. For patients with more than 2 year disease duration, the average number of attacks in the preceding 2 years will be calculated. For patients with less than 2 years disease duration the number of attacks in the preceding year will be used.
The safety profile of eculizumab in patients with NMO.
The secondary objectives are to determine:
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
- •Diagnosis of NMO, as defined by 2006 criteria OR NMO seropositive spectrum disorder (Recurrent ON or longitudinally extensive transverse myelitis (LETM)). All patients must be NMO-IgG seropositive.
- •Clinical evidence of at least 2 relapses in last 6 months or 3 relapses in the last 12 months (with at least 1 relapse occurring in the preceding 6 months).
- •Age ≥18 years
- •Corrected visual acuity 20/100 or better in at least one eye. If fails item # 4 then entry allowed but only if last attack was myelitis and only attacks of myelitis are considered as outcome measurement.
- •Ambulatory (with or without walker). If fails item # 5 then entry allowed but only if last attack was ON and only attacks of ON are considered as outcome measurement.
- •Provision of written informed consent (see attached) to participate in the study.
- •N. meningitidis vaccination at least 14 days prior to receiving the first eculizumab infusion. If patient in midst of an acute relapse, then relapse will be treated with standard therapy and vaccination given only after a minimum of 4 weeks post attack onset.
排除标准
- •Candidates will be excluded from study entry if any of the following criteria are met at the time of randomization:
- •Progressive neurological deterioration unrelated to relapses of ON or myelitis.
- •Pregnant, breastfeeding, or intending to conceive during the course of the study
- •Patients will not participate in any other clinical therapeutic study or will not have participated in any other experimental treatment study within 30 days of screening
- •Patients with a history of splenectomy, because of a potential increased risk of developing meningococcal infection.
研究组 & 干预措施
Eculizumab
The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks. Subjects will receive therapy for a total of 12 months.
干预措施: Eculizumab (Drug)
结局指标
主要结局
Median Number of Neuromyelitis Optica (NMO) Attacks Per Year
时间窗: baseline, after 12 months of treatment
次要结局
- Number Subjects Experiencing an NMO Attack in 12 Months of Eculizumab Treatment(12 months)
- Change in Expanded Disability Status Scale (EDDS) Score(baseline, 12 months)
- Percentage Hemolysis(baseline, 6 weeks, 3 months, 6 months, 9 months, 12 months)
- Mean Complement Protein 5 (C5) Concentration in CSF(baseline, 3 months)
- Number of Subjects With Change in Visual Acuity in at Least One Eye by at Least One Point(12 months)
- Number of Subjects With Change in Ambulation by at Least 1 Point(12 months)
- Mean Serum Concentration of Eculizumab(6 weeks, 3 months, 6 months, 9 months, 12 months)
- Mean Eculizumab Concentration in Cerebrospinal Fluid (CSF)(3 months)