A Randomized, Controlled, Open-label, Non Inferiority KawasakI Trial With Anakinra
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 38
- 主要终点
- Number of patients with treatment response in both treatment arms
研究概览
简要总结
This is a multicenter, open-label, randomized, controlled, interventional trial followed by a long-term observational extension period in patients with Kawasaki Disease (KD) to be treated eitherwith endovenous Immunoglobulins (IVIG-standard treatment) versus anakinra
Aim of the study: to demonstrate that anakinra is non-inferior to IVIG in KD, in terms of fever control in the acute phase and development of coronary artery dilation/aneurisms (CAA) within one year from the onset.
详细描述
This is a multicenter national, open label, randomized, controlled, interventional trial followed by a long-term observational extension period. This is a non-inferiority study Patients who fulfill the eligibility criteria and whose parent/carer (legal representative) has provided informed consent will be randomized 1:1 to receive either
- IVIG 2g/kg administered in 10-12 hours as per local standard of care (standard treatment) OR
- Anakinra 2mg/kg intravenously, max 100 mg/dose 4 times/day (investigational treatment)
PLUS Aspirin (ASA) 50mg/kg QID until 36 hours from fever disappearance, then switched to low-dose (3-5 mg/Kg once a day) as per standard of care
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
- 1 Month 至 16 Years（Child）
- 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
Anakinra
Anakinra 2mg/kg intravenously, max 100 mg/dose 4 times/day
干预措施: Anakinra (Drug)
Intravenous immunoglobulins
IVIG 2g/kg administered in 10-12 hours as per local standard of care
干预措施: Intravenous Immunoglobulins, Human (Drug)
结局指标
主要结局
Number of patients with treatment response in both treatment arms
时间窗: 12 months
Response rate
Number of patients with CAA (as per Z-scores) at the end of the study period in both treatment arms
时间窗: 12 months
CAA rate in both arms. CAAs will be classified in accordance with the scheme based on Z scores proposed by AHA. No coronary involvement with Z score \<2, dilation only with Z score \> 2 to \<2.5 or if initially \<2 with a decrease during follow-up ≥1 3, small aneurysm with Z score ≥2.5 to \<5, medium aneurysm with Z score ≥5 to \<10 and absolute dimension \<8 mm and large or giant aneurysm with Z score ≥10, or absolute dimension ≥8 mm
次要结局
- Number of days with fever in both treatment arms(90 days)
- Time to reach CRP values<50% from the highest value and to normalize it in both treatment arms (days)(90 days)
- Time to normalize coronary artery abnormalities (days)(90 days)
- Severity of coronary artery abnormalities (as per Z-score) at the end of follow-up(24 months)
- Length of hospitalization in both treatment arms (days)(90 days)
- Time to stop anakinra (days)(90 days)
- Adverse event and severe adverse event developed during the study and follow/up period(24 months)
- Cumulative drug exposure (mg/kg/day)(12 months)
研究者
Gabriele Simonini
Professor
Meyer Children's Hospital IRCCS