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Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease

This study has been terminated.
(data submitted for Food and Drug Administration (FDA) approval)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )
ClinicalTrials.gov Identifier:
NCT00069329
First received: September 22, 2003
Last updated: October 7, 2016
Last verified: October 2016
Results First Received: May 6, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Nervous System Malformations
Arthropathy, Neurogenic
Urticaria
Papilledema
Intervention: Drug: anakinra

  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
No text entered.

Reporting Groups
  Description
NOMID 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.

Participant Flow:   Overall Study
    NOMID
STARTED   43 
COMPLETED   41 
NOT COMPLETED   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
NOMID 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.

Baseline Measures
   NOMID 
Overall Participants Analyzed 
[Units: Participants]
 43 
Age 
[Units: Participants]
 
<=18 years   36 
Between 18 and 65 years   7 
>=65 years   0 
Gender 
[Units: Participants]
 
Female   25 
Male   18 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   8 
Not Hispanic or Latino   33 
Unknown or Not Reported   2 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   4 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   1 
White   29 
More than one race   1 
Unknown or Not Reported   8 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: Baseline ]

2.  Primary:   Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: 36 months ]

3.  Primary:   Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: 60 months ]

4.  Primary:   Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: Baseline ]

5.  Primary:   Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: 36 months ]

6.  Primary:   Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: 60 months ]

7.  Primary:   Parent /Patient Pain Rating   [ Time Frame: Baseline ]

8.  Primary:   Parent /Patient Pain Rating   [ Time Frame: 36 months ]

9.  Primary:   Parent /Patient Pain Rating   [ Time Frame: 60 months ]

10.  Primary:   Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: Baseline ]

11.  Primary:   Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: 36 months ]

12.  Primary:   Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: 60 months ]

13.  Primary:   Serum Amyloid A (SAA) Measurement   [ Time Frame: Baseline ]

14.  Primary:   Serum Amyloid A (SAA) Measurement   [ Time Frame: 36 months ]

15.  Primary:   Serum Amyloid A (SAA) Measurement   [ Time Frame: 60 months ]

16.  Primary:   C-reactive Protein (CRP) Measurement   [ Time Frame: Baseline ]

17.  Primary:   C-reactive Protein (CRP) Measurement   [ Time Frame: 36 months ]

18.  Primary:   C-reactive Protein (CRP) Measurement   [ Time Frame: 60 months ]

19.  Primary:   Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: Baseline ]

20.  Primary:   Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: 36 months ]

21.  Primary:   Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: 60 months ]


  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
Data reported is on 26 patients who completed their 3 year assessment and 20 of these patients also completed their 5 year assessment.


  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: Goldbach-Mansky, Raphaela
Organization: National Inst of Arthritis and Musculoskeletal and Skin Diseases
phone: +1 301 435 6243
e-mail: goldbacr@mail.nih.gov


Publications:
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 ( US NIH Grant/Contract Award Number )
03-AR-0298 ( Other Identifier: NIHCC )
Study First Received: September 22, 2003
Results First Received: May 6, 2016
Last Updated: October 7, 2016
Health Authority: United States: Federal Government
United States: Food and Drug Administration