CLINICAL TRIAL: STUDY OF RILONACEPT IN THE TREATMENT OF SYSTEMIC JUVENILE IDIOPA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 本研究的目的是严格测试IL-1抑制剂(利洛那普)对系统性幼年特发性关节炎(SJIA)关节和全身表现的短期安全性和有效性。该试验将利用一种新的研究设计，考虑到这种生物制剂预期的快速反应。治疗A组，在第0天给予利洛那普4.4 mg/kg至最大剂量320 mg，然后每周2.2 mg/kg至最大剂量160 mg，直到第4周再次给予负荷剂量，这次是安慰剂。在第二次负荷量之后，药物将在为期六个月的研究期间每周给药。治疗B组将以与治疗A组相同的方式接受安慰剂注射，然后在第4周接受负荷量的利洛那普，随后每周注射利洛那普，直到6个月的研究持续。这项试验将通过创建SJIA组织储存库来实现翻译研究，该组织储存库将在整个研究过程中定期收集DNA、RNA、外周血单核细胞、血清和血浆

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The goals of this study are to rigorously test the short-term safety and efficacy of an IL-1 inhibitor (rilonacept) on the articular and systemic manifestations of systemic juvenile idiopathic arthritis (SJIA). The trial will utilize a novel study design that takes into account the rapid onset of response expected with this biologic agent. Treatment Group A, will receive rilonacept at a dose of 4.4 mg/kg up to a maximum dose of 320 mg on day 0, and then weekly at a dose of 2.2mg/kg up to a maximum dose of 160 mg until week 4 when the loading dose is again given, this time with placebo. After the second loading dose, the drug will be administered weekly for the duration of the six-month study. Treatment Group B will get placebo injections in the same way as in Treatment Group A and then at week 4 will receive the loading dose of rilonacept, followed by weekly injections of rilonacept until the duration of the six month study. The trial will enable translational studies by creating a SJIA tissue repository that will bank DNA, RNA, peripheral blood mononuclear cells, serum and plasma collected at strategic intervals throughout the study