Safety and Effectiveness of Rilonacept for Treating Systemic Juvenile Idiopathic Arthritis in Children and Young Adults
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Randomized Placebo Phase Study of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis (RAPPORT)|
- Time to Response to Treatment, as Determined by a Modified JIA ACR30 Requiring no Fever, Coupled With a Requirement for Corticosteroid Taper in Participants Who Are Taking Corticosteroids [ Time Frame: At Week 12 ]
- Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS [ Time Frame: At Weeks 0- 24 ]
- Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70 [ Time Frame: At Week 4 and week 12 ]
- Pediatric Quality of Life Inventory [ Time Frame: At Weeks 4, 12 and 24 ]Visual Analog Score (0-100 mm) 0 very well , 100 very poor
- Physical Function as Determined by Childhood Health Assessment Questionnaire ( CHAQ) [ Time Frame: At Weeks 12 and 24 ]
Childhood Health Assesment Questionairre dissability index (C-HAQ)-DI, Disability Index Calculation:
The index is calculated by adding the scores for each of the categories and dividing by the number of categories answered. This gives a score in the 0 to 3.0 range. lower is better
- Number of Participants With Presence of Systemic Features ( Fever, Rash) [ Time Frame: At Weeks 4, 12 and 24 ]
|Study Start Date:||November 2008|
|Study Completion Date:||June 2013|
|Primary Completion Date:||June 2013 (Final data collection date for primary outcome measure)|
Experimental: Group 1
Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study
2.2 mg/kg subcutaneously
Placebo Comparator: Group 2
Placebo for 4 weeks, followed by rilonacept loading dose (4.4mg/kg), followed by rilonacept 2.2 mg/kg/week for the remainder of the study
2.2 mg/kg subcutaneously
The current standard treatment for SJIA includes nonsteroidal anti-inflammatory drugs (NSAIDS) and corticosteroids. However, in most people, NSAIDS do not completely control the disease. Also, no studies have been done to prove which medication or combination of medications is best to treat children and adolescents with SJIA. Interleukin-1 (IL-1), a protein secreted by certain cells in the body, assists in regulating immune and inflammatory responses. Too much IL-1 can be harmful and has been shown to play a role in the inflammation associated with a variety of diseases, including SJIA. Rilonacept is a drug that inhibits IL-1 activity. The purpose of this study is to determine whether a rilonacept drug regimen initiated early is more effective than a similar rilonacept drug regimen initiated 4 weeks later when treating children and young adults with SJIA. This study will also evaluate the safety of rilonacept, and various tissue samples will be collected from participants for future genetic studies.
This study will last 6 months. Participants will be randomly assigned to one of two groups:
- Group 1 participants will receive rilonacept injections at a dose of 4.4mg/kg at study entry (loading dose), then 2.2 mg/kg weekly until Week 4. At Week 4, they will receive a loading dose of placebo, followed by weekly rilonacept injections at 2.2 mg/kg for the duration of the study.
- Group 2 participants will receive placebo at study entry and then during the first 4 weeks of treatment. At Week 4, they will receive a loading dose of rilonacept injections of 4.4 mg/kg, followed by weekly rilonacept injections at a dose of 2.2 mg/kg for the duration of the study.
Participants will continue any previous corticosteroid therapy, but in tapering doses. All participants will attend study visits at Weeks 0, 2, 4, 6, 8, 10, 12, 14 and 24. Study visits will include a physical exam, joint exam, blood collection, interview, and questionnaires. Urine collection may occur for some female participants. Other evaluations may be performed by the participant's regular doctor. Throughout the study, participants will maintain at-home diaries to record fever, morning stiffness and pain, when rilonacept or placebo was taken, any side effects experienced from treatment, and any additional medications that were taken.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00534495
|United States, New York|
|Montefiore Medical Center|
|Bronx, New York, United States, 10467|
|Principal Investigator:||Norman T. Ilowite, MD||Montefiore Medical Center|