Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders (BEAT NMO)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- The number of attacks
研究概览
简要总结
Neuromyelitis Optica Spectrum Disorders (NMOSD) is associated with a pathological humoral immune response against the aquaporin-4(AQP-4) water channel. Belimumab (Benlysta ®) is a human immunoglobulin G1λ monoclonal antibody that inhibits B-cell survival and differentiation by neutralizing soluble B lymphocyte stimulator. Belimumab may benefit some patients with NMOSD due to the important role of B cells in the pathogenesis of NMOSD. Clinical trials may be needed to observe its efficacy and safety.
详细描述
The investigators primarily aim to observe the number of attacks from initiation of belimumab treatment.
The secondary outcomes are to determine: The safety profile of belimumab in participants with NMO and whether belimumab improves Expanded Disability Status Scale (EDSS), et al.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 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
- 是
入选标准
- •Male or female patients ≥ 18 years old
- •Diagnosis of NMO or NMO spectrum disorder according to the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria
- •Clinical evidence of either at least one attack requiring rescue therapy (intravenous corticosteroids, intravenous immunoglobulin, plasma exchange, or a combination of these therapies) in the year before screening or at least two attacks requiring rescue therapy in the 2 years before screening.
- •EDSS <= 6.0
- •Patients were seropositive for AQP4-IgG
- •Able and willing to give written informed consent and comply with the requirements of the study protocol.
排除标准
- •Current evidence or known history of clinically significant infection (Herpes simplex virus, varicella-zoster virus, cytomegalovirus, Epstein-Barr virus, human immunodeficiency virus, Hepatitis viruses, Syphilis, etc)
- •Participation in another interventional trial within the last 3 months
- •Tumor disease currently or within last 5 years
- •Pregnant, breastfeeding, or child-bearing potential during the course of the study
- •Clinically relevant heart, liver, kidney or bone marrow function disorder
研究组 & 干预措施
Belimumab
Belimumab will be intravenously administered with a dose of 10mg/kg on Days 0,14 and28, then every 28 days until week 48, with a final evaluation at week 52.
干预措施: Belimumab (Drug)
结局指标
主要结局
The number of attacks
时间窗: From baseline to one year after
An acute attack was defined as a new neurological worsening lasting for at least 24 hours and occurring more than 30 days after the previous attack.
次要结局
- Number of New, and/or Enlarging T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)(From baseline to 52 weeks)
- Determination of serum AQP4 antibodies(From baseline to 52 weeks)
- Worsening in EDSS(Worsening from baseline in EDSS to 52 weeks)
- Incidence of treatment-emergent adverse events [safety and tolerability](From baseline to 52 weeks)
- Counts of peripheral blood B cell subsets(From baseline to 52 weeks)
研究者
Qiang Liu
Professor of Department of Neurology
Tianjin Medical University General Hospital