Clinical Trial: Safety and Effectiveness of Rilonacept for Treating Systemic Juvenile Idiopathic Arthritis in Children and Young Adults

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Randomized Placebo Phase Study of Rilonacept in the Treatment of Systemic Juvenile Idiopathic Arthritis (RAPPORT)

Brief Summary: Systemic juvenile idiopathic arthritis (SJIA) is a type of arthritis that typically occurs before 16 years of age. SJIA usually involves heat, pain, swelling, and stiffness in the body's joints. It can also involve fever, rash, anemia, and inflammation in various parts of the body. Rilonacept is a drug that can reduce inflammation. 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.

Detailed Summary:

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
Sponsor: Montefiore Medical Center

Current Primary Outcome:

  • 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 ]


Original Primary Outcome: Response to treatment, as determined by JIA ACR30 [ Time Frame: At Weeks 12 and 24 ]

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome:

  • Response to treatment, as determined by JIA ACR50 and JIA ACR70 [ Time Frame: At Weeks 12 and 24 ]
  • Pediatric Quality of Life Inventory [ Time Frame: At Weeks 12 and 24 ]
  • Physical function as determined by Childhood Health Assessment Questionnaire [ Time Frame: At Weeks 12 and 24 ]
  • Presence of systemic features [ Time Frame: At Weeks 12 and 24 ]


Information By: Montefiore Medical Center

Dates:
Date Received: September 24, 2007
Date Started: November 2008
Date Completion:
Last Updated: November 5, 2015
Last Verified: November 2015