NCT06660329Enrolling By Invitation4 期
Efficacy and Safety of Tofacitinib in Patients with Refractory Blau Syndrome: a Prospective Cohort Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- Enrolling By Invitation
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- rate of remission or low disease activity
研究概览
简要总结
This is a prospective cohort study to observe the efficacy and safety of Tofacitinib in children with Blau syndrome (BS). The investigators would analyze the rate of remission or low disease activity after treatment as well as changes in inflammatory markers, patients' and physician's global assessment of disease activity to determine the efficacy and safety of Tofacitinib.
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
- 0 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have pathogenic mutation(s) in NOD2 gene;
- •Patients who have clinical manifestations such as granulomatous dermatitis, arthritis, uveitis, vasculitis, interstitial lung disease and so on;
- •Clinical remission was not achieved after ≥12 weeks of treatment with at least one immunosuppressant or biologics.
排除标准
- •Patients will not be included if meets any of the following criteria:
- •Being treated with IL-1 inhibitor, or other biological agents;
- •Pregnant and lactating women;
- •Serious organ function failure, expected life time less than 6 months.
研究组 & 干预措施
Janus kinase inhibitors
Experimental
Tofacitinib is used according to weight: 5~<7kg，2mg；7~<10kg，2.5mg；10~<15kg，3mg；15~<25kg，3.5mg；25~<40kg，4mg；≥40kg，5mg. All is twice a day.
干预措施: Janus Kinase Inhibitor (Drug)
结局指标
主要结局
rate of remission or low disease activity
时间窗: From enrollment to the end of treatment at 6 months
次要结局
- RCB(Response in Chinese children with Blau syndrome) 30, 50, 70 response rates(From enrollment to the end of treatment at 3, 6, 9, 12 months)
- Changes in inflammatory markers (including erythrocyte sedimentation rate, C reactive protein), cytokines (including IL-1β, IL-6, IL-17, IL-18, TNFα, IFN γ) and expression of type I interferon-stimulated genes over baseline(From enrollment to the end of treatment at 1,3, 6, 9, 12 months)
- Proportion of recurrent uveitis(From enrollment to the end of treatment at 12 months)
- Incidence of new organ involvement(From enrollment to the end of treatment at 1,3, 6, 9, 12 months)
- Number of participants with adverse effect(From enrollment to the end of treatment at 12 months)
研究者
Hongmei Song
Professor
Peking Union Medical College Hospital
研究点 (1)
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