Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 71
- 试验地点
- 1
- 主要终点
- Changes in Auto-Inflammatory Diseases Activity Index score after 6-month treatment over baseline
研究概览
简要总结
This is a prospective cohort study to observe the efficacy and safety of tranilast in CAPS patients. The investigators would analyze the changes in Auto-Inflammatory Diseases Activity Index (AIDAI) before and 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 tranilast.
详细描述
Seventy-one patients with CAPS will be recruited. After signing the informed consent, they will be administrated with tranilast (For juvenile patients, 5mg/kg.d with a maximum dose of 0.3g per day; For adult patients, the dose is 0.1g each time, three times a day). These patients will be followed up for 6 months. AIDAI is recorded by patients' or their parents one month before the start of treatment, and at the 1st, 3rd and 6th month after the treatment. Inflammatory markers, and patients' and physician's global assessment of disease activity will be assessed during the 1st, 3rd and 6th month follow-up. Side effects will be monitored and recorded as well. Experimental data before and after the administration of tranilast will be analyzed and be statistically processed, to figure out whether tranilast is effective and safe for CAPS patients.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients must meet the following diagnostic criteria of CAPS and have pathogenic mutation(s) in NLRP3 gene.
- •Raised inflammatory markers (CRP/SAA) (mandatory criteria)
- •≥2 of 6 CAPS typical signs/symptoms:
- •Urticaria-like rash;
- •Cold/stress triggered episodes;
- •Sensorineural hearing loss;
- •Musculoskeletal symptoms (arthralgia/arthritis/myalgia);
- •Chronic aseptic meningitis;
- •Skeletal abnormalities (epiphyseal overgrowth/frontal bossing).
排除标准
- •Patients will not be included if meets any of the following criteria:
- •Being treated with IL-1 inhibitor, other biological agents and immunosuppressants
- •Pregnant and lactating women
- •Serious organ function failure, expected life time less than 6 months
研究组 & 干预措施
Tranilast
5mg/kg.d for juvenile patients with a maximum dose of 0.3g per day; 0.1g each time, three times a day for adults patients
干预措施: Tranilast (Drug)
结局指标
主要结局
Changes in Auto-Inflammatory Diseases Activity Index score after 6-month treatment over baseline
时间窗: The previous 1 month before treatment and the 6th month after treatment
Patients or their parents completed a 1-month (31 days) prospective diary with 12 yes/no items( Fever ≥38°C, Overall symptoms, Abdominal pain, Nausea/vomiting, Diarrhoea, Headaches, Chest pain, Painful nodes, Arthralgia or myalgia, Swelling of the joints, Eye manifestations, Skin rash) at the previous 1 month before treatment, and the 6th month after treatment . Each item of this diary was dichotomised as no (0)=absence of symptom or yes (1)=presence of symptom. The calculation of the Auto-Inflammatory Diseases Activity Index score is straightforward, consisting of the sum of all 12 items (0-372 in a month of 31 days). Higher values represent higher disease activity.
次要结局
- Changes in Auto-Inflammatory Diseases Activity Index score at the 1st and 3rd month over baseline(The previous 1 month before treatment and the 1st and 3rd month after treatment)
- Changes in inflammatory markers, including C-reactin protein, erythrocyte sedimentation rate, serum amyloid protein, interleukin-1β and interleukin-18， at 1, 3 and 6 months over baseline(Baseline and at 1, 3 and 6 months after treatment)
- Changes in physician global assessment of disease activity on a 0-10 visual analog scale (VAS) at 1, 3 and 6 months over baseline(Baseline and 1, 3 and 6 months after treatment)
- Changes in parent/patient global assessment of well-being on a 0-10 visual analogue score (VAS) at 1, 3 and 6 months over baseline(Baseline and 1, 3 and 6 months after treatment)
- Changes in CSF white blood cell count for CINCA patients(Baseline and 6 months after treatment.)
- Changes in MRI of the brain and inner ear for CINCA patients(Baseline and 6 months after treatment.)
- Changes in audiology data for CINCA patients(Baseline and 6 months after treatment.)
- Number of participants with adverse effect(Up to 6 months)
研究者
Hongmei Song
Professor
Peking Union Medical College Hospital