A Randomized, Double Blind, Placebo Controlled Study of the Effects of Etanercept in Children Presenting With Kawasaki Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 205
- 试验地点
- 8
- 主要终点
- IVIG Refractory
研究概览
简要总结
The purpose of this study is to determine whether Etanercept (Enbrel) when used in conjunction with IVIG and aspirin, improves treatment response to IVIG in patients with Kawasaki Disease. Funding Source- FDA/OOPD
详细描述
Kawasaki Disease (KD) is a potentially life threatening acute vasculitis in children with a predilection for involvement of the coronary arteries. Aspirin and Intravenous gamma globulin (IVIG) are principally used for the treatment of the symptoms of Kawasaki Disease. Aspirin reduces inflammation and platelet formation, but has no effect in attenuating the development of coronary abnormalities. Although IVIG reduces inflammation and the prevalence of coronary artery abnormalities, it has a relatively high failure rate of 23-30%, warranting new treatment methods for Kawasaki Disease. We propose a placebo controlled double blinded randomized study to determine if etanercept 0.8 mg/kg subcutaneously (max 25 mg) given three times at weekly intervals starting at initial diagnosis is safe in this patient population and if it is a successful adjunct therapy with IVIG in reducing the incidence of persistent or recurrent fever.
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 Months 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male Age 2 months to 20 years of age Female Age 2 months to 11 years of age
- •Provision of Parental Consent
- •Kawasaki Disease Presentation
排除标准
- •Laboratory Criteria: Any laboratory toxicity, at the time of the screening visit or at any time during the study that in the opinion of the Investigator would preclude participation in the study or:
- •Platelet count < 100,000/mm3
- •WBC count < 3,000 cells/mm3
- •Hemoglobin, hematocrit, or red blood cell count outside 30% of the upper or lower limits of normal for the Lab
- •Subject is currently enrolled in another investigational device or drug trial(s), or subject has received other investigational agent(s) within 28 days of baseline visit.
- •Female subjects diagnosed with KD 12 years of age and older.
- •Subjects who have known hypersensitivity to Enbrel or any of its components or who is known to have antibodies to etanercept
- •Prior or concurrent cyclophosphamide therapy
- •Prior treatment with any TNF alpha antagonist or steroid within 48 hours prior to initiation of IVIG
- •Concurrent sulfasalazine therapy
- •Active severe infections within 4 weeks before screening visit, or between the screening and baseline visits.
- •SLE, history of multiple sclerosis, transverse myelitis, optic neuritis, or chronic seizure disorder
- •Known HIV-positive status or known history of any other immuno-suppressing disease.
- •Any mycobacterial disease or high risk factors for tuberculosis, such as family member with TB or taking INH
- •Untreated Lyme disease
- •Severe comorbidities (diabetes mellitus requiring insulin, CHF of any severity, MI, CVA or TIA within 3 months of screening visit, unstable angina pectoris, uncontrolled hypertension (sitting systolic BP > 160 or diastolic BP > 100 mm Hg), oxygen-dependent severe pulmonary disease, history of cancer within 5 years [other than resected cutaneous basal or squamous cell carcinoma or in situ cervical cancer])
- •Exposure to hepatitis B or hepatitis C or high risk factors such as intravenous drug abuse in patient's mother, or history of jaundice (other than neonatal jaundice). SLE, history of multiple sclerosis, transverse myelitis, optic neuritis or chronic seizure disorder.
- •Use of a live vaccine (Measles Mumps Rubella or Varicella) 30 days prior to or during this study.
- •Any condition judged by the patient's physician to cause this clinical trial to be detrimental to the patient
- •History of non-compliance with other therapies
- •Must not have received immunosuppressive agents for at least three months prior to enrollment.
研究组 & 干预措施
Arm 1 -Etanercept
Drug - Treatment with Etanercept as adjunct to standard treatment with IVIG and aspirin
干预措施: Etanercept (Drug)
2
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
IVIG Refractory
时间窗: 42 days after initial dose
The primary outcome is the proportion of subjects who become refractory to IVIG. Subjects requiring 1 dose of IVIG are classified as responders and subjects requiring more than 1 dose are classified as IVIG refractory.
次要结局
- Determine if Etanercept Treatment Alters the Rate of Coronary Artery Dilation and Disease (CAD) at 2 and 6 Weeks After Treatment in Patients With Dilated Coro(42 days after initial dose)
研究者
Michael Portman
Cardiologist
Seattle Children's Hospital