TOLERABILITY AND PRELIMINARY EFFICACY STUDY OF TWO DOSE LEVELS OF IL-1 TRAP

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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. Systemic Juvenile Idiopathic Arthritis (S-JIA) represents about 10 to 20% of all cases of JIA in children. The cause of JIA is unknown. At this time, there is no optimal therapy for children with S-JIA. Interleukin-1 (IL-1) has been shown to be a key inflammatory molecule involved in active S-JIA. This is a Phase I pilot study to determine an appropriate dose of IL-1 Trap (a new weekly anti-IL-1 drug), evaluate the safety of IL-1 Trap, and its effectiveness in improving the disease manifestations in children with S-JIA. This is a study that will be performed in up to 10 centers and enroll 18 subjects between the ages of 4-21 with the diagnosis of S-JIA. The first 9 subjects enrolled will receive either weekly injections of either 2.2 mg/kg IL-1 Trap (6 subjects) or placebo (3 subjects) for 4 weeks. The injections are given into the tissue just under the skin (subcutaneous) like insulin injections. After evaluation of the initial safety data, if the 2.2 mg/kg dose appears to be safe, the next 9 subjects will be enrolled and will receive either 4.4 mg/kg IL-1 Trap (6 subjects) or placebo (3 subjects) for 4 weeks. During the 4 weeks of the study neither the subjects of the clinic staff will know who is getting the IL-1 Trap or placebo (a blinded study). The decision about who will receive the IL-1 Trap or placebo will be done by random selection. After completion of the 4 weeks of the blinded study, all participants will receive treatment with open label IL-1 Trap for 13 months. During this study, blood will be drawn to measure levels of IL-1 Trap to show the peak level and how quickly it is eliminated from children with Systemic JIA. This information will be used to see if the IL-1 Trap levels are similar in adult and children given IL-1 Trap. In addition, information from this study may be used in planning for a larger study in children with Systemic JIA.