Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2, Open Label Trial (EASE-NMO)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 75
- 主要终点
- Mean change in OSIS from presentation to Day 28
研究概览
简要总结
Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.
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
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
IVMP arm
IVMP arm: 1000mg methylprednisolone x5d, oral prednisone 60mg, 5mg weekly decline + antibiotics
干预措施: IVMP (Drug)
eculizumab arm
Eculizumab arm: eculizumab (900 mg) will be administered intravenously once per week for a total of four doses (days 1, 8, 15, and 22) in conjunction with IVMP and oral prednisone (60mg, 5mg weekly decline). Terminal complement complex inhibition by eculizumab predisposes patients to infections by encapsulated bacteria, especially N meningitidis. Meningococcal vaccination will not be effective in this timeframe; instead, all enrolled patients will receive antibiotic prophylaxis against N meningitidis from the time of the first dose of the study drug to 8 weeks after the last administration
干预措施: Complement protein C5 inhibitor (Drug)
eculizumab arm
Eculizumab arm: eculizumab (900 mg) will be administered intravenously once per week for a total of four doses (days 1, 8, 15, and 22) in conjunction with IVMP and oral prednisone (60mg, 5mg weekly decline). Terminal complement complex inhibition by eculizumab predisposes patients to infections by encapsulated bacteria, especially N meningitidis. Meningococcal vaccination will not be effective in this timeframe; instead, all enrolled patients will receive antibiotic prophylaxis against N meningitidis from the time of the first dose of the study drug to 8 weeks after the last administration
干预措施: IVMP (Drug)
结局指标
主要结局
Mean change in OSIS from presentation to Day 28
时间窗: Acute attack to week4
The Optic-Spinal Impairment Score (OSIS) was developed to measure the disability status of subjects with demyelinating disease. It allows an objective quantification of the level of functioning that could be widely and reproducibly used by researchers and health care providers. The OSIS evaluates four primary functions: Visual Acuity (VA) (0-8), Motor Function (0-7), Sensory Function (0-5), and Sphincter Function (0-5). A higher OSIS score indicates a more severe level of disability. The standards for the OSIS scoring are as follows: Optic-Spinal Impairment Score (OSIS) Visual Acuity (VA) 0 Normal 1. Scotoma but VA (corrected) better than 20/30 2. VA 20/30-20/59 3. VA 20/60-20/100 4. VA 20/I0I-2012004 5. VA 20/20I-20/800. 6. Count fingers only 7. Light perception only 8. No light perception Motor Function 0 Normal 1. Abnormal signs (hyperreflexia, Babinski sign) without weakness 2. Mild weakness (MRC grade 5-or 4+) in affected limb(s) 3. Mo
次要结局
- Change in EDSS from presentation to Day 28(Acute attack to week4)
研究者
Fu-Dong Shi
Professor
Tianjin Medical University General Hospital