A Randomized, Double-blind, Placebo Controlled, Single-dose Study to Assess the Initial Efficacy of Canakinumab (ACZ885) With Respect to the Adapted ACR Pediatric 30 Criteria in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Active Systemic Manifestations
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 84
- 试验地点
- 17
- 主要终点
- Percentage of Patients Who Meet the Adapted American College of Rheumatology (ACR) Pediatric 30 Criteria
研究概览
简要总结
This study assessed the initial efficacy and safety of canakinumab over a 4 week period in patients with systemic juvenile idiopathic arthritis (SJIA) having a flare. Response to treatment will be according to the adapted American College of Rheumatology(ACR)Pediatric 30 criteria at Day 15.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 19 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of systemic juvenile idiopathic arthritis as per ILAR definition that must have occurred at least 2 months prior to enrollment with onset of disease < 16 years of age:
- •Arthritis in one or more joints with or preceded by fever of at least 2 weeks duration that is documented to be daily/quotidian for at least 3 days and accompanied by one or more of the following:
- •evanescent nonfixed erythematous rash,
- •generalized lymph node enlargement,
- •hepatomegaly and/ or splenomegaly,
- •serositis
- •Parent's or legal guardian's written informed consent and child's assent, if appropriate, or patient's informed consent for ≥ 18 years of age
- •Male and female patients aged ≥ 2 to < 20 years of age
- •Active disease at the time of enrollment defined as follows:
- •At least 2 joints with active arthritis
- •Documented spiking, intermittent fever (body temperature > 38°C) for at least 1 day during the screening period within 1 week before first canakinumab/placebo dose
- •C-reactive protein (CRP) > 30 mg/L (normal range < 10 mg/L)
- •Naïve to canakinumab
- •Other protocol defined inclusion criteria may apply
排除标准
- •Patients who fulfilled one or more of the following criteria were not eligible for inclusion in this study:
- •Pregnant or nursing (lactating) female patients
- •Female patients having reached sexual maturity unless their career, lifestyle, or sexual orientation precluded intercourse with a male partner and/or they were using an acceptable method of contraception
- •History of hypersensitivity to study drug or to biologics.
- •Diagnosis of active macrophage-activation syndrome (MAS) (Ravelli, Magni-Manzoni and Pistorio 2005) within the last 6 months
- •With active or recurrent bacterial, fungal or viral infection, including patients with evidence of human immunodeficiency virus (HIV) infection, hepatitis B or hepatitis C infection
- •Other protocol defined exclusion criteria may apply
研究组 & 干预措施
Canakinumab
Patients received a single dose of subcutaneous(sc) injection of canakinumab (4 mg/kg) on Day 1. Maximal total single dose of canakinumab allowed was 300 mg. Any patient who required a dose greater than 150 mg (patients>37.5 kg) received two sc injections.
干预措施: Canakinumab (Drug)
Placebo
Patients received a single dose matching placebo of canakinumab on day 1.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Patients Who Meet the Adapted American College of Rheumatology (ACR) Pediatric 30 Criteria
时间窗: Baseline, Day 15, Day 29
Adapted ACR Pediatric 30 criteria determined responders (improved from baseline of at least 30% in at least 3 response variables 1-6 and no intermittent fever in preceding week \[variable 7\], with no more than one variable 1-6 worsening \> 30% ) 1. Physician's Global Assessment of disease activity: 0-100 mm VAS 2.Parent/Patient's Global Assessment of Patient's overall wellbeing: 0-100mmVAS in Child Health Assessment Questionnaire (CHAQ) 3. Functional ability: CHAQ 4.Number of joints with active arthritis 5. Number of joints with limited of motion 6. Laboratory measure of inflammation CRP (mg/L)
次要结局
- Percentage of Patients Achieving the Adapted ACR Pediatric 50 Criteria(Baseline, Day 15, Day 29)
- Percentage of Patients Achieving the Adapted ACR Pediatric 70(Baseline, Day 15, Day 29)
- Percentage of Patients Achieving the Adapted ACR Pediatric 90(Baseline, Day 15, Day 29)
- Percentage of Patients Achieving the Adapted ACR Pediatric 100(baseline, Day 15, Day 29)
- Change in Patient's Pain Intensity as Assessed on a 100-mm Visual Analog Scale (VAS)as Part of the Childhood Health Assessment Questionnaire(CHAQ)(Baseline, Day 15)
- Change in Patient's Pain Intensity as Assessed on a 100-mm Visual Analog Scale (VAS) as Part of CHAQ(Baseline, Day 29)
- Percentage of Patients Who Had Body Temperature ≤ 38°C(Day 3)
- Change in Health-related Quality of Life (HRQoL)Over Time by Use of the Child Health Questionnaire - Parent Form (CHQ-PF50)(Over 4 week study period (Baseline, Day 15, Day 29))
- Change in Disability Score Over Time by Use of the CHAQ(At 4 week study period)