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

This study has been completed.
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)
ClinicalTrials.gov Identifier:
NCT00534495
First received: September 24, 2007
Last updated: November 5, 2015
Last verified: November 2015
Results First Received: November 5, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Juvenile Idiopathic Arthritis
Intervention: Biological: Rilonacept

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
71 participants enrolled. 1 participant was erroneously randomized and was not included in the analysis. This patient was not exposed to study drug

Reporting Groups
  Description
Rilonacept Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week followed by a placebo loading dose and then rilonacept in the long term extension phase Rilonacept: 2.2 mg/kg subcutaneously
Placebo

Placebo loading dose followed by maintenance dose for 4 weeks, followed by rilonacept loading dose (4.4mg/kg), followed by rilonacept 2.2 mg/kg/week for the long term extension

Rilonacept: 2.2 mg/kg subcutaneously

Long Term Extension All Participants All participants who benefited from rilonacept were eligible to enroll this phase and receive rilonacept 2.2mg/kg weekly

Participant Flow for 3 periods

Period 1:   Double Blind Placebo Phase Week 0-4
    Rilonacept     Placebo     Long Term Extension All Participants  
STARTED     36     35     0  
COMPLETED     36     34     0  
NOT COMPLETED     0     1     0  
randomized in error                 0                 1                 0  

Period 2:   All Active Treatment Phase Week 4-24
    Rilonacept     Placebo     Long Term Extension All Participants  
STARTED     36     34     0  
COMPLETED     32     25     0  
NOT COMPLETED     4     9     0  
Adverse Event                 1                 0                 0  
Lack of Efficacy                 1                 7                 0  
Lost to Follow-up                 1                 1                 0  
Withdrawal by Subject                 1                 1                 0  

Period 3:   Long Term Ext. Phase Week 24-month 21
    Rilonacept     Placebo     Long Term Extension All Participants  
STARTED     0     0     40  
COMPLETED     0     0     29  
NOT COMPLETED     0     0     11  
Adverse Event                 0                 0                 1  
Lack of Efficacy                 0                 0                 5  
Withdrawal by Subject                 0                 0                 3  
non compliance                 0                 0                 2  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Rilonacept

Loading dose of rilonacept (4.4mg/kg) at Week 0, followed by rilonacept 2.2 mg/kg/week for the remainder of the study

Rilonacept: 2.2 mg/kg subcutaneously

Placebo

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

Rilonacept: 2.2 mg/kg subcutaneously

Total Total of all reporting groups

Baseline Measures
    Rilonacept     Placebo     Total  
Number of Participants  
[units: participants]
  36     35     71  
Age  
[units: years]
Mean (Standard Deviation)
  9.5  (4.6)     10.5  (4.4)     10  (4.5)  
Gender  
[units: participants]
     
Female     23     23     46  
Male     13     12     25  
Race/Ethnicity, Customized [1]
[units: Participants]
     
Black     5     7     12  
White     25     23     48  
Other     6     5     11  
Hispanic     7     5     12  
Non-Hispanic     29     30     59  
Disease Duration  
[units: years]
Mean (Standard Deviation)
  2.6  (3.6)     2.6  (3.1)     2.6  (3.4)  
No.of joints with active disease  
[units: joints]
Mean (Standard Deviation)
  11.7  (9.6)     10.5  (7.6)     11.1  (8.6)  
Prior medications  
[units: participants]
     
Corticosteroids     30     33     63  
Methotrexate     21     26     47  
Leflunomide     1     2     3  
Infliximab     5     6     11  
Etanercept     12     16     28  
Abatacept     5     4     9  
Anakinra     13     13     26  
unknown Anakinra     4     4     8  
Disease characteristics in the past  
[units: participants]
     
Incomplete Macrophage Activation Syndrome     1     3     4  
Complete Macrophage Activation Syndrome     1     1     2  
Serositis     9     8     17  
Systemic JIA rash     32     33     65  
[1] Black ,white and Other are races Hispanic and Non-Hispanic are ethnicities. All patients were counted twice.



  Outcome Measures
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1.  Primary:   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 ]

2.  Primary:   Number of Serious Adverse Events,Adverse Events, Infections, Development of MAS   [ Time Frame: At Weeks 0- 24 ]

3.  Secondary:   Number of Participants With Response as Determined by JIA ACR50 and JIA ACR70   [ Time Frame: At Week 4 and week 12 ]

4.  Secondary:   Pediatric Quality of Life Inventory   [ Time Frame: At Weeks 4, 12 and 24 ]

5.  Secondary:   Physical Function as Determined by Childhood Health Assessment Questionnaire ( CHAQ)   [ Time Frame: At Weeks 12 and 24 ]

6.  Secondary:   Number of Participants With Presence of Systemic Features ( Fever, Rash)   [ Time Frame: At Weeks 4, 12 and 24 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr.Norman T.Ilowite
Organization: Children's Hospital at Montefiore
phone: 718-696-2602
e-mail: NILOWITE@montefiore.org



Responsible Party: Norman Ilowite, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
ClinicalTrials.gov Identifier: NCT00534495     History of Changes
Other Study ID Numbers: N01 AR070015
268200700015C-2-0-0 ( US NIH Grant/Contract Award Number )
HHSN2682007000015C
Study First Received: September 24, 2007
Results First Received: November 5, 2015
Last Updated: November 5, 2015
Health Authority: United States: Food and Drug Administration