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Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease

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ClinicalTrials.gov Identifier: NCT00069329
Recruitment Status : Terminated (data submitted for Food and Drug Administration (FDA) approval)
First Posted : September 23, 2003
Results First Posted : December 1, 2016
Last Update Posted : December 1, 2016
Sponsor:
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
Conditions Nervous System Malformations
Arthropathy, Neurogenic
Urticaria
Papilledema
Intervention Drug: anakinra
Enrollment 43
Recruitment Details  
Pre-assignment Details  
Arm/Group Title NOMID
Hide Arm/Group Description Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Period Title: Overall Study
Started 43
Completed 41
Not Completed 2
Arm/Group Title NOMID
Hide Arm/Group Description Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Baseline Participants 43
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 43 participants
<=18 years
36
  83.7%
Between 18 and 65 years
7
  16.3%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 43 participants
Female
25
  58.1%
Male
18
  41.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 43 participants
Hispanic or Latino
8
  18.6%
Not Hispanic or Latino
33
  76.7%
Unknown or Not Reported
2
   4.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 43 participants
American Indian or Alaska Native
0
   0.0%
Asian
4
   9.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   2.3%
White
29
  67.4%
More than one race
1
   2.3%
Unknown or Not Reported
8
  18.6%
1.Primary Outcome
Title Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)
Hide Description "The severity of the main symptoms of the disease were scored on a scale from 0 (no symptoms) to 4 (highest severity) on a daily basis using a diary. Five key symptoms were included in the primary variable DSSS: fever, headache, rash, joint pain, and vomiting. Each of the diary variables was evaluated as a mean value for a period preceding the visits. The baseline value was the mean value of the 5-30 last days before the first dose of Kineret. For the subsequent visits, the mean value of the last 30 days with data before each visit was used as the response variable."
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.75741904  (0.08370955)
2.Primary Outcome
Title Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)
Hide Description "The severity of the main symptoms of the disease were scored on a scale from 0 (no symptoms) to 4 (highest severity) on a daily basis using a diary. Five key symptoms were included in the primary variable DSSS: fever, headache, rash, joint pain, and vomiting. Each of the diary variables was evaluated as a mean value for a period preceding the visits. The baseline value was the mean value of the 5-30 last days before the first dose of Kineret. For the subsequent visits, the mean value of the last 30 days with data before each visit was used as the response variable."
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.08704545  (0.02470396)
3.Primary Outcome
Title Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)
Hide Description "The severity of the main symptoms of the disease were scored on a scale from 0 (no symptoms) to 4 (highest severity) on a daily basis using a diary. Five key symptoms were included in the primary variable DSSS: fever, headache, rash, joint pain, and vomiting. Each of the diary variables was evaluated as a mean value for a period preceding the visits. The baseline value was the mean value of the 5-30 last days before the first dose of Kineret. For the subsequent visits, the mean value of the last 30 days with data before each visit was used as the response variable."
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.10768421  (0.04158048)
4.Primary Outcome
Title Patient / Parent Global Score of Overall Disease Activity
Hide Description Visual analog assessment of how arthritis affects the patient as rated by the patient themselves or parent. Measured on scale from very well (0 mm) to very poor (100 mm).
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
40.22381  (4.6242215)
5.Primary Outcome
Title Patient / Parent Global Score of Overall Disease Activity
Hide Description Visual analog assessment of how arthritis affects the patient as rated by the patient themselves or parent. Measured on scale from very well (0 mm) to very poor (100 mm).
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
8.1923077  (2.7291978)
6.Primary Outcome
Title Patient / Parent Global Score of Overall Disease Activity
Hide Description Visual analog assessment of how arthritis affects the patient as rated by the patient themselves or parent. Measured on scale from very well (0 mm) to very poor (100 mm).
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
11.1  (3.6322025)
7.Primary Outcome
Title Parent /Patient Pain Rating
Hide Description Visual analog assessment of how much pain the patient experienced in past week due to illness as rated by the patient themselves or parent. Measured on scale from no pain (0 mm) to very severe pain (100 mm).
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
41.22381  (4.9988185)
8.Primary Outcome
Title Parent /Patient Pain Rating
Hide Description Visual analog assessment of how much pain the patient experienced in past week due to illness as rated by the patient themselves or parent. Measured on scale from no pain (0 mm) to very severe pain (100 mm).
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
8.9423077  (2.892075)
9.Primary Outcome
Title Parent /Patient Pain Rating
Hide Description Visual Analog assessment of how much pain the patient experienced in past week due to illness as rated by the patient themselves or parent. Measured on scale from no pain (0 mm) to very severe pain (100 mm).
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
9.8525  (3.1635745)
10.Primary Outcome
Title Childhood Health Assessment Questionnaire (CHAQ)
Hide Description Patient self-assessment (or parent assessment) 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).
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
1.1051429  (0.21122155)
11.Primary Outcome
Title Childhood Health Assessment Questionnaire (CHAQ)
Hide Description Patient self-assessment (or parent assessment) 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).
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.66346154  (0.15898857)
12.Primary Outcome
Title Childhood Health Assessment Questionnaire (CHAQ)
Hide Description Patient self-assessment (or parent assessment) 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).
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.590625  (0.180202)
13.Primary Outcome
Title Serum Amyloid A (SAA) Measurement
Hide Description Serum Amyloid A is an inflammatory marker for NOMID measured using Rapid Automated Enzyme Immunoassay. Normal SAA values were defined as ≤ 10 mg/liter.
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mg/liter
220.28077  (38.27684)
14.Primary Outcome
Title Serum Amyloid A (SAA) Measurement
Hide Description Serum Amyloid A is an inflammatory marker for NOMID measured using Rapid Automated Enzyme Immunoassay. Normal SAA values were defined as ≤ 10 mg/liter.
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mg/liter
22.333333  (7.5122284)
15.Primary Outcome
Title Serum Amyloid A (SAA) Measurement
Hide Description Serum Amyloid A is an inflammatory marker for NOMID measured using Rapid Automated Enzyme Immunoassay. Normal SAA values were defined as ≤ 10 mg/liter.
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: mg/liter
21.3125  (11.586169)
16.Primary Outcome
Title C-reactive Protein (CRP) Measurement
Hide Description C-reactive protein (CRP) is an inflammatory marker for NOMID. Systemic inflammatory remission was defined as a normal CRP level (≤0.5mg/dl). Analyzed at the NIH Clinical Center Laboratory.
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mg/dl
57.703077  (7.2213655)
17.Primary Outcome
Title C-reactive Protein (CRP) Measurement
Hide Description C-reactive protein (CRP) is an inflammatory marker for NOMID. Systemic inflammatory remission was defined as a normal CRP level (≤0.5mg/dl). Analyzed at the NIH Clinical Center Laboratory.
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mg/dl
8.7511538  (2.2674947)
18.Primary Outcome
Title C-reactive Protein (CRP) Measurement
Hide Description C-reactive protein (CRP) is an inflammatory marker for NOMID. Systemic inflammatory remission was defined as a normal CRP level (≤0.5mg/dl). Analyzed at the NIH Clinical Center Laboratory.
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: mg/dl
6.0585  (2.3462251)
19.Primary Outcome
Title Erythrocyte Sedimentation Rate (ESR) Measurement
Hide Description Erythrocyte Sedimentation Rate (ESR) is an inflammatory marker for NOMID. Normal ESR values are defined as ≤ 25 mm/hour. Analyzed at the NIH Clinical Center Laboratory.
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg/day per injection were made as frequently as every 2 weeks to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mm/hour
56.923077  (6.3138244)
20.Primary Outcome
Title Erythrocyte Sedimentation Rate (ESR) Measurement
Hide Description Erythrocyte Sedimentation Rate (ESR) is an inflammatory marker for NOMID. Normal ESR values are defined as ≤ 25 mm/hour. Analyzed at the NIH Clinical Center Laboratory.
Time Frame 36 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 4.5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 26 patients who completed their 3 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: mm/hour
11.576923  (1.3222045)
21.Primary Outcome
Title Erythrocyte Sedimentation Rate (ESR) Measurement
Hide Description Erythrocyte Sedimentation Rate (ESR) is an inflammatory marker for NOMID. Normal ESR values are defined as ≤ 25 mm/hour. Analyzed at the NIH Clinical Center Laboratory.
Time Frame 60 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients started at 1mg/kg by daily subcutaneous injection. Stepwise dose increases of 0.5-1 mg/kg per injection were made as frequently as every 2 weeks up to 5 mg/kg/day to achieve laboratory and organ inflammation remission. Data reported is on 20 patients who completed their 5 year assessment.
Arm/Group Title NOMID
Hide Arm/Group Description:
Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: mm/hour
11.35  (2.4355103)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title NOMID
Hide Arm/Group Description Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.
All-Cause Mortality
NOMID
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
NOMID
Affected / at Risk (%)
Total   18/43 (41.86%) 
Cardiac disorders   
hospital admission for cardiac catheterization   1/43 (2.33%) 
Eye disorders   
Uveitis   1/43 (2.33%) 
Gastrointestinal disorders   
diarrhea   1/43 (2.33%) 
Gastritis   1/43 (2.33%) 
vomiting   1/43 (2.33%) 
Immune system disorders   
Cytokine release syndrome   1/43 (2.33%) 
Infections and infestations   
Acute Tonsillitis/tooth abscess   1/43 (2.33%) 
Appendicitis   1/43 (2.33%) 
Arthritis   1/43 (2.33%) 
Cellulitis Right lower extremity   1/43 (2.33%) 
Left cervical lymphadenitis   1/43 (2.33%) 
Lung infection   2/43 (4.65%) 
Meningitis   1/43 (2.33%) 
Pneumonitis   1/43 (2.33%) 
Right ankle cellulitis   1/43 (2.33%) 
Wound infection   1/43 (2.33%) 
Metabolism and nutrition disorders   
Metabolic toxicity   1/43 (2.33%) 
Musculoskeletal and connective tissue disorders   
generalized muscle weakness   1/43 (2.33%) 
Pain   1/43 (2.33%) 
Nervous system disorders   
Headache   5/43 (11.63%) 
Meningitis   1/43 (2.33%) 
Pain   1/43 (2.33%) 
Seizure   1/43 (2.33%) 
Renal and urinary disorders   
Renal calculi   1/43 (2.33%) 
Reproductive system and breast disorders   
Double Mastectomy   1/43 (2.33%) 
Low grade focal ductal carcinoma in-situ of left breast   1/43 (2.33%) 
Right breast inflammation post surgery   1/43 (2.33%) 
Right breast scar/fibrotic tissue excised from inplant capsule   1/43 (2.33%) 
Skin and subcutaneous tissue disorders   
Hospitalized for Rash   1/43 (2.33%) 
Surgical and medical procedures   
Bilateral Capsulectomy with permenant implants   1/43 (2.33%) 
L ureter stent placed   1/43 (2.33%) 
Left renal stent removal   1/43 (2.33%) 
Lithotripsy   1/43 (2.33%) 
Post-Surgery Pneumonia   1/43 (2.33%) 
Suicidal ideation   1/43 (2.33%) 
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
NOMID
Affected / at Risk (%)
Total   43/43 (100.00%) 
Blood and lymphatic system disorders   
bicarbonate   1/43 (2.33%) 
blood / Bone Marrow - Other - elevated Hematocrit   1/43 (2.33%) 
blood / Bone Marrow - Other - eosinophilia   2/43 (4.65%) 
blood / Bone Marrow - Other - low reticulocyte count   1/43 (2.33%) 
Blood bilirubin increased   1/43 (2.33%) 
Bruising   4/43 (9.30%) 
Epistaxis   4/43 (9.30%) 
hemoglobin   1/43 (2.33%) 
Hypomagnesemia   1/43 (2.33%) 
Hyponatremia   1/43 (2.33%) 
Proteinuria   1/43 (2.33%) 
Transient elevation of LFT's   1/43 (2.33%) 
Cardiac disorders   
Angiodema Right ring finger   1/43 (2.33%) 
Chest wall pain   4/43 (9.30%) 
Hypertension   1/43 (2.33%) 
Palpitations   1/43 (2.33%) 
Sinus tachycardia   1/43 (2.33%) 
Vasovagal reaction   2/43 (4.65%) 
Ear and labyrinth disorders   
ear drainage   1/43 (2.33%) 
ear pain   1/43 (2.33%) 
Hearing impaired   2/43 (4.65%) 
Tinnitus   1/43 (2.33%) 
Endocrine disorders   
Hypothyroidism   1/43 (2.33%) 
weight gain   2/43 (4.65%) 
Eye disorders   
Conjunctivitis   16/43 (37.21%) 
Dry eye   1/43 (2.33%) 
Eye infection   1/43 (2.33%) 
eye inflammation (left)   1/43 (2.33%) 
Eye pain   4/43 (9.30%) 
eye throbbing   1/43 (2.33%) 
foreign body in eye   1/43 (2.33%) 
Glaucoma   1/43 (2.33%) 
hemmorage in eye   1/43 (2.33%) 
itchy eyes   1/43 (2.33%) 
Nystagmus   2/43 (4.65%) 
Pain in extremity   1/43 (2.33%) 
sty on left eye   1/43 (2.33%) 
vision - eye twitch   1/43 (2.33%) 
Gastrointestinal disorders   
Abdominal pain   7/43 (16.28%) 
Alanine aminotransferase increased   2/43 (4.65%) 
Anorexia   1/43 (2.33%) 
Aspartate aminotransferase increased   1/43 (2.33%) 
canker sore   1/43 (2.33%) 
Cold Sore on Gums   1/43 (2.33%) 
Constipation   4/43 (9.30%) 
Diarrhea   10/43 (23.26%) 
Dysphagia   1/43 (2.33%) 
Flatulence   1/43 (2.33%) 
Gastritis   6/43 (13.95%) 
Gastrointestinal pain   6/43 (13.95%) 
Increased Liver Enzymes   1/43 (2.33%) 
Nausea   6/43 (13.95%) 
Uveitis   1/43 (2.33%) 
vomiting   9/43 (20.93%) 
Weight loss   1/43 (2.33%) 
General disorders   
Disease Flare   1/43 (2.33%) 
Fatigue   7/43 (16.28%) 
Flare Symptoms (pt had 1 week of no anakinra)   1/43 (2.33%) 
Flare, ran out of anakinra   1/43 (2.33%) 
Insomnia   7/43 (16.28%) 
Irritability   1/43 (2.33%) 
Malaise   2/43 (4.65%) 
NOMID Flare   1/43 (2.33%) 
Immune system disorders   
Allergic reaction   2/43 (4.65%) 
Allergic rhinitis   1/43 (2.33%) 
Infections and infestations   
Allergic rhinitis   3/43 (6.98%) 
Blastocystis Hominis Infection   1/43 (2.33%) 
bowel abcess   1/43 (2.33%) 
Bronchial infection   5/43 (11.63%) 
Candida on palms and soles of feet bilaterally   1/43 (2.33%) 
C-Diff Recurrence   1/43 (2.33%) 
Chest Infection (?Pneumonia)   1/43 (2.33%) 
Chest wall pain   1/43 (2.33%) 
Chills   1/43 (2.33%) 
Conjunctivitis   4/43 (9.30%) 
Croup   1/43 (2.33%) 
ear drainage   1/43 (2.33%) 
Ear pain   1/43 (2.33%) 
Eczema / Atopic Dermatitis   1/43 (2.33%) 
External ear inflammation   14/43 (32.56%) 
Fever   16/43 (37.21%) 
Flu like symptoms   15/43 (34.88%) 
Folliculitis - L-Groin   1/43 (2.33%) 
G Tube Infection   1/43 (2.33%) 
Gastritis   7/43 (16.28%) 
Gastrointestinal pain   1/43 (2.33%) 
Hand-Foot-Mouth Disease   1/43 (2.33%) 
Headache   1/43 (2.33%) 
Hearing impaired   2/43 (4.65%) 
Hypopyon - Left eye   1/43 (2.33%) 
infected finger   1/43 (2.33%) 
infection without Neutropenia   1/43 (2.33%) 
infection without Neutropenia - (yeast)   1/43 (2.33%) 
infection without Neutropenia - Left arm thrombophlebitis   1/43 (2.33%) 
infection without Neutropenia - tonsillitis with sore throat   1/43 (2.33%) 
infection without Neutropenia (tooth abcess)   1/43 (2.33%) 
itchy   1/43 (2.33%) 
Lice   1/43 (2.33%) 
Lung infection   3/43 (6.98%) 
Mouth Infection   1/43 (2.33%) 
mouth sores   1/43 (2.33%) 
Nasal congestion   9/43 (20.93%) 
Oral Ulcer   1/43 (2.33%) 
Otitis externa   2/43 (4.65%) 
Otitis media   10/43 (23.26%) 
Pharyngitis   3/43 (6.98%) 
Pneumonitis   1/43 (2.33%) 
R ear D/C   1/43 (2.33%) 
Red & burning sensation on bottom of feet   1/43 (2.33%) 
ringworm   1/43 (2.33%) 
Rotaviral Infection   1/43 (2.33%) 
RSV (Resp. Syncytial Virus)   1/43 (2.33%) 
RSV infection   1/43 (2.33%) 
Scalp Lesion (? Tinea capitis)   1/43 (2.33%) 
scarlett fever   1/43 (2.33%) 
Shingles   1/43 (2.33%) 
Sinusitis   15/43 (34.88%) 
Skin infection   1/43 (2.33%) 
sore throat   10/43 (23.26%) 
Strep throat   2/43 (4.65%) 
strep throat (2x)   1/43 (2.33%) 
Stye   1/43 (2.33%) 
Throat infection   1/43 (2.33%) 
Tooth infection (?abscess)   1/43 (2.33%) 
Upper respiratory infection   19/43 (44.19%) 
Urinary tract infection   4/43 (9.30%) 
Urinary tract pain   5/43 (11.63%) 
Viral Illness   2/43 (4.65%) 
Viral Illness Dehydration   1/43 (2.33%) 
Viral Infection   4/43 (9.30%) 
Viral Infection Fever, Lethargy   1/43 (2.33%) 
Vision loss   1/43 (2.33%) 
yeast infection   1/43 (2.33%) 
Injury, poisoning and procedural complications   
Auto Accident   1/43 (2.33%) 
Fall   7/43 (16.28%) 
Physical Assault   1/43 (2.33%) 
Investigations   
CPK increased   1/43 (2.33%) 
Metabolism and nutrition disorders   
Weight gain   1/43 (2.33%) 
Musculoskeletal and connective tissue disorders   
Anxiety   1/43 (2.33%) 
Arthralgia   16/43 (37.21%) 
Back pain   7/43 (16.28%) 
Chest wall pain   1/43 (2.33%) 
Conjunctivitis   1/43 (2.33%) 
Dysmenorrhea   1/43 (2.33%) 
Edema limbs   1/43 (2.33%) 
Fatigue   5/43 (11.63%) 
Flu like symptoms   2/43 (4.65%) 
Fracture   3/43 (6.98%) 
Gait disturbance   4/43 (9.30%) 
Malaise   1/43 (2.33%) 
Myalgia   3/43 (6.98%) 
Myositis   1/43 (2.33%) 
Neck pain   7/43 (16.28%) 
Osgood-Schlatter   1/43 (2.33%) 
pain   6/43 (13.95%) 
Pain in extremity   14/43 (32.56%) 
restless leg syndrome   1/43 (2.33%) 
S/P L Knee growth plate fixation/fever thematoma   1/43 (2.33%) 
Nervous system disorders   
A.D.D.   1/43 (2.33%) 
Allergic reaction   1/43 (2.33%) 
Anxiety   1/43 (2.33%) 
Blurred vision   2/43 (4.65%) 
carpal tunnel - right   1/43 (2.33%) 
cerebrospinal fluid leakage   1/43 (2.33%) 
Dizziness   4/43 (9.30%) 
External ear pain   1/43 (2.33%) 
Fever   1/43 (2.33%) 
Generalized muscle weakness   1/43 (2.33%) 
Glaucoma   1/43 (2.33%) 
Headache   23/43 (53.49%) 
Hearing impaired   3/43 (6.98%) 
Hoarseness   1/43 (2.33%) 
Nausea   1/43 (2.33%) 
Neck pain   1/43 (2.33%) 
Pain in extremity   1/43 (2.33%) 
Paresthesia of R hand suggestive of carpel tunnel   1/43 (2.33%) 
Saccadic Pursuit Neuropathy - Cranial   1/43 (2.33%) 
Seizure   1/43 (2.33%) 
Uveitis   1/43 (2.33%) 
Vertigo   3/43 (6.98%) 
vomiting   1/43 (2.33%) 
Renal and urinary disorders   
Bladder infection   2/43 (4.65%) 
Catheter related infection   1/43 (2.33%) 
Flank pain   1/43 (2.33%) 
glucosuria   1/43 (2.33%) 
Hematuria   1/43 (2.33%) 
Ketonuria   1/43 (2.33%) 
metabolic (elevated BUN)   1/43 (2.33%) 
metabolic / Laboratory - Other - elevated Blood Urea Nitrogen   1/43 (2.33%) 
Proteinuria   1/43 (2.33%) 
Renal calculi   2/43 (4.65%) 
Renal colic   1/43 (2.33%) 
Upper respiratory infection   2/43 (4.65%) 
Urinary incontinence   1/43 (2.33%) 
Urinary tract infection   3/43 (6.98%) 
Urinary tract pain   2/43 (4.65%) 
Reproductive system and breast disorders   
Breakthrough bleeding (menstrual)   1/43 (2.33%) 
Phantom pain of breats bilateraly   1/43 (2.33%) 
Respiratory, thoracic and mediastinal disorders   
Bronchospasm   1/43 (2.33%) 
Cough   9/43 (20.93%) 
Dyspnea   2/43 (4.65%) 
Gait disturbance   1/43 (2.33%) 
Nasal congestion   2/43 (4.65%) 
Sinusitis   1/43 (2.33%) 
Stridor   1/43 (2.33%) 
Skin and subcutaneous tissue disorders   
Allergic reaction   2/43 (4.65%) 
bug bite   1/43 (2.33%) 
cyst on Right cheek   1/43 (2.33%) 
Dry skin   3/43 (6.98%) 
Eczema (contact dermatitis)   1/43 (2.33%) 
eczematous reaction to injection   1/43 (2.33%) 
Erythema Left Foot   1/43 (2.33%) 
eye swollen   1/43 (2.33%) 
fungal rash   1/43 (2.33%) 
hive   1/43 (2.33%) 
hives   1/43 (2.33%) 
Hot/diaphoretic in summer months - rash at beltline   1/43 (2.33%) 
Ingrown toenail   1/43 (2.33%) 
Injection site reaction   10/43 (23.26%) 
Intermittent rash on body   1/43 (2.33%) 
lesion on scrotum   1/43 (2.33%) 
Oral Ulcers   2/43 (4.65%) 
Papulopustular rash   1/43 (2.33%) 
Penile pain   1/43 (2.33%) 
Perineal pain   1/43 (2.33%) 
R. 2nd toenail injury with ingrown nail   1/43 (2.33%) 
Rash   13/43 (30.23%) 
Rash - (contact dermatitis)   1/43 (2.33%) 
Rash - adhesive (IV site)   1/43 (2.33%) 
Rash - Bugbite - R ear   1/43 (2.33%) 
Rash - Lichenified rash on glutial crack   1/43 (2.33%) 
Rash - Reaction to U-bag   1/43 (2.33%) 
Rash (viral infection)   1/43 (2.33%) 
rash / desquamation   1/43 (2.33%) 
Rash arms, trunk, legs   1/43 (2.33%) 
Rash on Arms   1/43 (2.33%) 
Rash on Right Leg   1/43 (2.33%) 
rashes   1/43 (2.33%) 
red cheeks   1/43 (2.33%) 
Red Dots   1/43 (2.33%) 
red dots - rash   1/43 (2.33%) 
Red Dots on Arm   1/43 (2.33%) 
Skin infection   1/43 (2.33%) 
Surgical and medical procedures   
Cesarean Section   1/43 (2.33%) 
Dental Surgery Outpatient   1/43 (2.33%) 
Elective L cochlear implant surgery   1/43 (2.33%) 
Elective Osteotomy   1/43 (2.33%) 
Endoscopy to replace G Tube   1/43 (2.33%) 
eye surgery   1/43 (2.33%) 
hand surgery - carpal tunnel   1/43 (2.33%) 
Kidney Stent removal   1/43 (2.33%) 
Lithotripsy/stent removed   1/43 (2.33%) 
Placement of BMT (tubes)   1/43 (2.33%) 
staples in knee (surgery)   1/43 (2.33%) 
Surgery Adenoidectomy, Placement of bilateral myringotomy tubes   1/43 (2.33%) 
surgery for cyst removal   1/43 (2.33%) 
surgery on right knee   1/43 (2.33%) 
surgery on wrist   1/43 (2.33%) 
Tonsillectomy   1/43 (2.33%) 
Ureteral stent for renal calculi - Right kidney   1/43 (2.33%) 
Wisdom Teeth Extraction   1/43 (2.33%) 
Vascular disorders   
hypertension   1/43 (2.33%) 
Indicates events were collected by systematic assessment
Data reported is on 26 patients who completed their 3 year assessment and 20 of these patients also completed their 5 year assessment.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Goldbach-Mansky, Raphaela
Organization: National Inst of Arthritis and Musculoskeletal and Skin Diseases
Phone: +1 301 435 6243
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )
ClinicalTrials.gov Identifier: NCT00069329     History of Changes
Other Study ID Numbers: 030298
ZIAAR041138-08 ( U.S. NIH Grant/Contract )
03-AR-0298 ( Other Identifier: NIHCC )
First Submitted: September 22, 2003
First Posted: September 23, 2003
Results First Submitted: May 6, 2016
Results First Posted: December 1, 2016
Last Update Posted: December 1, 2016