Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 11
- 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. Baricitinib is an oral Janus kinase (JAK)1/JAK2 inhibitor that blocks the upregulated JAK-STAT pathway in patients with neuroimmune disorders, which is important in bone marrow regulation of B cell proliferation and differentiation. Baricitinib 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 relapses from initiation of baricitinib treatment.
The secondary outcomes are to determine: The safety profile of baricitinib in participants with NMO and whether baricitinib 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
- 否
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 relapse requiring rescue therapy (intravenous corticosteroids, intravenous immunoglobulin, plasma exchange or a combination of these therapies) in the year before screening or at least two relapses requiring rescue therapy in the 2 years before screening;
- •Expanded disability status scale (EDSS) score ≤ 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 study within the last 3 months;
- •Tumor disease currently or within the last 5 years;
- •Pregnancy, breastfeeding, or child-bearing potential during the course of the study;
- •Patients with clinically relevant heart, liver, kidney or bone marrow dysfunction;
- •History of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.
研究组 & 干预措施
Baricitinib
Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96.
干预措施: Baricitinib (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
The number of relapses
时间窗: From baseline to 96 weeks
A relapse was defined as new-onset neurological symptoms or worsening of existing neurological function (vision loss, limb weakness or sensory symptoms, or bladder or bowel dysfunction) lasting more than 24 h, not attributable to an identifiable cause such as intercurrent infection, and preceded by at least 30 days of clinical stability.
次要结局
- Changes in serum AQP4 antibodies(From baseline to 52 weeks)
- Changes in peripheral blood B cell subsets(From baseline to 52 weeks)
- Changes in EDSS(Changes in EDSS from baseline to 52 weeks)
- Changes in the 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)
- Incidence of treatment-emergent adverse events [safety and tolerability](From baseline to 52 weeks)
- Changes in EDSS scores(Changes in EDSS from baseline to 96 weeks)
- Changes in the number of new and/or enlarging lesions on T2-weighted imaging (T2WI) and gadolinium-enhancing lesions on T1-weighted imaging (T1WI).(From baseline to 96 weeks)
- Changes in the number of peripheral blood B cell subsets(From baseline to 96 weeks)
- Changes in serum AQP4-IgG titer(From baseline to 96 weeks)
- Incidence of treatment-emergent adverse events [safety and tolerability](From baseline to 96 weeks)
研究者
Qiang Liu
Professor of Department of Neurology
Tianjin Medical University General Hospital