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Trial record 1 of 5 for:    dira
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Rilonacept for Deficiency of the Interleukin-1 Receptor Antagonist (DIRA)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01801449
First Posted: February 28, 2013
Last Update Posted: October 16, 2017
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.
Collaborator:
National Institutes of Health Clinical Center (CC)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )
Results First Submitted: August 8, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: DIRA
Intervention: Drug: 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
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Reporting Groups
  Description
Rilonacept Treatment Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week.Total duration of the study 24 months.

Participant Flow:   Overall Study
    Rilonacept Treatment
STARTED   6 
COMPLETED   6 
NOT COMPLETED   0 



  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 Treatment Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.

Baseline Measures
   Rilonacept Treatment 
Overall Participants Analyzed 
[Units: Participants]
 6 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      6 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      3  50.0% 
Male      3  50.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      4  66.7% 
Not Hispanic or Latino      2  33.3% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      0   0.0% 
White      3  50.0% 
More than one race      0   0.0% 
Unknown or Not Reported      3  50.0% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Subjects Who Maintained Inflammatory Remission With Rilonacept   [ Time Frame: 6 months ]

2.  Secondary:   Height   [ Time Frame: 24 months after rilonacept initiation ]

3.  Secondary:   Height   [ Time Frame: Baseline ]

4.  Secondary:   Weight   [ Time Frame: 24 months after rilonacept initiation ]

5.  Secondary:   Weight   [ Time Frame: Baseline ]

6.  Secondary:   Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: 24 months after rilonacept initiation ]

7.  Secondary:   Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: Baseline ]

8.  Secondary:   Dual-energy X-ray Absorptiometry (DEXA) Scores   [ Time Frame: 12 months after rilonacept initiation ]

9.  Secondary:   Dual-energy X-ray Absorptiometry (DEXA) Scores   [ Time Frame: Baseline ]

10.  Secondary:   Pediatric Quality of Life Inventory (PedsQL)   [ Time Frame: 24 months after rilonacept initiation ]

11.  Secondary:   Pediatric Quality of Life Inventory (PedsQL)   [ Time Frame: Baseline ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Montealegre, Gina
Organization: National Inst of Arthritis and Musculoskeletal and Skin Diseases
phone: +1 301 761 7747
e-mail: montealegrega@mail.nih.gov


Publications of Results:
Other Publications:

Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )
ClinicalTrials.gov Identifier: NCT01801449     History of Changes
Other Study ID Numbers: 130086
13-AR-0086 ( Other Identifier: The National Institutes of Health )
First Submitted: February 27, 2013
First Posted: February 28, 2013
Results First Submitted: August 8, 2017
Results First Posted: October 16, 2017
Last Update Posted: October 16, 2017