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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00534495
Recruitment Status : Completed
First Posted : September 26, 2007
Results First Posted : December 11, 2015
Last Update Posted : December 11, 2015
Sponsor:
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
Norman Ilowite, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Tracking Information
First Submitted Date  ICMJE September 24, 2007
First Posted Date  ICMJE September 26, 2007
Results First Submitted Date  ICMJE November 5, 2015
Results First Posted Date  ICMJE December 11, 2015
Last Update Posted Date December 11, 2015
Study Start Date  ICMJE November 2008
Actual Primary Completion Date June 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 5, 2015)
  • 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 Measures  ICMJE
 (submitted: September 24, 2007)
Response to treatment, as determined by JIA ACR30 [ Time Frame: At Weeks 12 and 24 ]
Change History Complete list of historical versions of study NCT00534495 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2015)
  • 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 Measures  ICMJE
 (submitted: September 24, 2007)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Effectiveness of Rilonacept for Treating Systemic Juvenile Idiopathic Arthritis in Children and Young Adults
Official Title  ICMJE 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 Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Juvenile Idiopathic Arthritis
Intervention  ICMJE Biological: Rilonacept
2.2 mg/kg subcutaneously
Other Names:
  • IL-1 Trap
  • Arcalyst
Study Arms  ICMJE
  • 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
    Intervention: Biological: Rilonacept
  • 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
    Intervention: Biological: Rilonacept
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 15, 2014)
71
Original Estimated Enrollment  ICMJE
 (submitted: September 24, 2007)
100
Actual Study Completion Date  ICMJE June 2013
Actual Primary Completion Date June 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Fulfills International League Against Rheumatism (ILAR) criteria for SJIA
  • Duration of SJIA lasting at least 6 weeks since onset
  • Active disease as defined by at least two joints with active disease
  • Not currently receiving methotrexate OR if taking methotrexate, the dose has remained stable or has been discontinued for 4 weeks prior to screening
  • Has never received certain biologics OR if previously received biologics, discontinued etanercept for at least 4 weeks prior to screening and discontinued infliximab or adalimumab for at least 8 weeks prior to screening
  • Not currently receiving corticosteroids OR if taking oral corticosteroids, the dose has remained stable between 2 and 60 mg/day for at least 2 weeks prior to screening

Exclusion Criteria:

  • Past treatment with anakinra, rilonacept, or other biologic IL-1 inhibitor
  • Treatment with other disease-modifying antirheumatic drugs (DMARDs) including, but not limited to, azathioprine, sulfasalazine, cyclosporine, and thalidomide within 4 weeks of screening
  • Treatment with leflunomide without cholestyramine washout at the end of therapy
  • Treatment with cyclophosphamide within 3 months of study entry
  • Treatment with tacrolimus or tocilizumab within 4 weeks of study entry
  • Treatment with rituximab within 6 months of study entry
  • Treatment with intravenous immunoglobulin (IVIG) within 4 weeks of screening
  • Kidney disease
  • AST or ALT levels more than two times the upper limit of normal
  • Bilirubin levels higher than 1.5 mg/dl
  • Thrombocytopenia, leukopenia, or neutropenia
  • Abnormal prothrombin time (PT) and partial thromboplastin time (PTT) tests
  • Low levels of plasma fibrinogen
  • Evidence of chronic recurrent infection or other significant, non-SJIA illness that might interfere with study participation
  • Psychological or cognitive difficulties that might interfere with study participation
  • Current drug or alcohol abuse
  • Anticipated poor compliance to assigned study regimen
  • Participation in another clinical trial within 30 days of study entry
  • Major surgical procedure within 3 months of study entry
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Months to 19 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00534495
Other Study ID Numbers  ICMJE N01 AR070015
268200700015C-2-0-0 ( U.S. NIH Grant/Contract )
HHSN2682007000015C
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Norman Ilowite, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Study Sponsor  ICMJE Montefiore Medical Center
Collaborators  ICMJE National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators  ICMJE
Principal Investigator: Norman T. Ilowite, MD Montefiore Medical Center
PRS Account Montefiore Medical Center
Verification Date November 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP