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Rilonacept for Deficiency of the Interleukin-1 Receptor Antagonist (DIRA)

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ClinicalTrials.gov Identifier: NCT01801449
Recruitment Status : Completed
First Posted : February 28, 2013
Results First Posted : October 16, 2017
Last Update Posted : October 16, 2017
Sponsor:
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) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition DIRA
Intervention Drug: Rilonacept
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 6
Completed 6
Not Completed 0
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
6
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
3
  50.0%
Male
3
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Hispanic or Latino
4
  66.7%
Not Hispanic or Latino
2
  33.3%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 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%
1.Primary Outcome
Title Number of Subjects Who Maintained Inflammatory Remission With Rilonacept
Hide Description Inflammatory remission criteria remission criteria were defined as meeting all of the following criteria: a diary score of <0.5 (reflecting no fever, skin rash or bone pain), normal acute phase reactants (C-reactive protein (CRP) <0.5 mg/dL), no objective skin rash or radiological evidence of active bone lesions on x-ray.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
6
 100.0%
2.Secondary Outcome
Title Height
Hide Description Antropometric measurements - Height z-score
Time Frame 24 months after rilonacept initiation
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-1.22  (0.48)
3.Secondary Outcome
Title Height
Hide Description Antropometric measurements - Height z-score
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-1.1  (0.41)
4.Secondary Outcome
Title Weight
Hide Description Antropometric measurements - Weight z-score
Time Frame 24 months after rilonacept initiation
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-0.48  (0.64)
5.Secondary Outcome
Title Weight
Hide Description Antropometric measurements - Weight z-score
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-0.59  (0.62)
6.Secondary Outcome
Title Childhood Health Assessment Questionnaire (CHAQ)
Hide Description The Childhood Health Assessment Questionnaire (CHAQ) is a patient self-assessment (or parent assessment) tool for how illness affects ability to function in daily life. Includes overall score, overall pain rating, overall global evaluation, subcategories for dressing and grooming, arising, eating, walking, hygiene, reach, grip, and activities. Measured on scale of 0-3 from 'Without any difficulty' (0) to 'Unable to do' (3). The scores are summed and divided by the number of total categories answered to arrive at a final value.
Time Frame 24 months after rilonacept initiation
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Units on a scale
0.25  (0.38)
7.Secondary Outcome
Title Childhood Health Assessment Questionnaire (CHAQ)
Hide Description The Childhood Health Assessment Questionnaire (CHAQ) is a patient self-assessment (or parent assessment) tool for how illness affects ability to function in daily life. Includes overall score, overall pain rating, overall global evaluation, subcategories for dressing and grooming, arising, eating, walking, hygiene, reach, grip, and activities. Measured on scale of 0-3 from 'Without any difficulty' (0) to 'Unable to do' (3). The scores are summed and divided by the number of total categories answered to arrive at a final value.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Units on a scale
0.83  (0.74)
8.Secondary Outcome
Title Dual-energy X-ray Absorptiometry (DEXA) Scores
Hide Description Mean z-scores of anteroposterior lumbar (AP) spine were used for comparisons
Time Frame 12 months after rilonacept initiation
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-0.12  (1.26)
9.Secondary Outcome
Title Dual-energy X-ray Absorptiometry (DEXA) Scores
Hide Description Mean z-scores of anteroposterior lumbar (AP) spine were used for comparisons
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: z-score
-0.08  (1.23)
10.Secondary Outcome
Title Pediatric Quality of Life Inventory (PedsQL)
Hide Description Pediatric Quality of Life Inventory (PedsQL)-assess functional impairment and change in treatment and health-related quality of life respectively. Responses on this scale are transformed into a 0-100 scale, with 0 being the worst value for health-related quality of life and 100 being the best possible value for health-related quality of life.
Time Frame 24 months after rilonacept initiation
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Units on a scale
90.6  (4.85)
11.Secondary Outcome
Title Pediatric Quality of Life Inventory (PedsQL)
Hide Description Pediatric Quality of Life Inventory (PedsQL)-assess functional impairment and change in treatment and health-related quality of life respectively. Responses on this scale are transformed into a 0-100 scale, with 0 being the worst value for health-related quality of life and 100 being the best possible value for health-related quality of life.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis included all subjects
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Units on a scale
86.6  (8.51)
Time Frame 24 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Rilonacept Treatment
Hide Arm/Group Description 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.
All-Cause Mortality
Rilonacept Treatment
Affected / at Risk (%)
Total   0/6 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Rilonacept Treatment
Affected / at Risk (%)
Total   0/6 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Rilonacept Treatment
Affected / at Risk (%)
Total   6/6 (100.00%) 
Blood and lymphatic system disorders   
Thrombocytosis   1/6 (16.67%) 
Ear and labyrinth disorders   
Otitis Externa   1/6 (16.67%) 
Otitis Media   4/6 (66.67%) 
Eye disorders   
Conjunctivitis allergic   1/6 (16.67%) 
Gastrointestinal disorders   
Abdominal pain   2/6 (33.33%) 
Constipation   1/6 (16.67%) 
Gastroenteritis   2/6 (33.33%) 
Pharyngitis   3/6 (50.00%) 
Vomiting   2/6 (33.33%) 
General disorders   
Pyrexia   2/6 (33.33%) 
Immune system disorders   
Allergy to chemicals   1/6 (16.67%) 
Food allergy   1/6 (16.67%) 
Skin reaction   1/6 (16.67%) 
Infections and infestations   
Pneumonia   1/6 (16.67%) 
Sinusitis   1/6 (16.67%) 
Upper respiratory tract infection   6/6 (100.00%) 
Investigations   
Blood creatine phosphokinase increased   2/6 (33.33%) 
C-reactive protein increased   1/6 (16.67%) 
Metabolism and nutrition disorders   
Hypercholesterolaemia   1/6 (16.67%) 
Hyperkalaemia   1/6 (16.67%) 
Musculoskeletal and connective tissue disorders   
Arthralgia   2/6 (33.33%) 
Myalgia   1/6 (16.67%) 
Nervous system disorders   
Headache   2/6 (33.33%) 
Respiratory, thoracic and mediastinal disorders   
Rhinorrhoea   3/6 (50.00%) 
Upper respiratory tract congestion   3/6 (50.00%) 
Skin and subcutaneous tissue disorders   
Dry skin   1/6 (16.67%) 
Eczema   1/6 (16.67%) 
Photosensitivity reaction   1/6 (16.67%) 
Pruritis   1/6 (16.67%) 
Rash   5/6 (83.33%) 
Seborrhoeic dermatitis   1/6 (16.67%) 
Indicates events were collected by systematic assessment
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
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