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

History of Changes

Results First Received: May 6, 2016

Study Type:	Interventional
Study Design:	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
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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
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.
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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

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1. Primary:

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

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2. Primary:

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

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3. Primary:

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

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4. Primary:

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

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5. Primary:

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

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6. Primary:

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

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7. Primary:

Parent /Patient Pain Rating [ Time Frame: Baseline ]

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8. Primary:

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

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9. Primary:

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

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10. Primary:

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

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11. Primary:

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

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12. Primary:

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

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13. Primary:

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

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14. Primary:

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

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15. Primary:

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

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16. Primary:

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

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17. Primary:

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

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18. Primary:

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

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19. Primary:

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

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20. Primary:

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

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21. Primary:

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

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Serious Adverse Events

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Other Adverse Events

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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:

Feldmann J, Prieur AM, Quartier P, Berquin P, Certain S, Cortis E, Teillac-Hamel D, Fischer A, de Saint Basile G. Chronic infantile neurological cutaneous and articular syndrome is caused by mutations in CIAS1, a gene highly expressed in polymorphonuclear cells and chondrocytes. Am J Hum Genet. 2002 Jul;71(1):198-203. Epub 2002 May 24.

Hashkes PJ, Lovell DJ. Recognition of infantile-onset multisystem inflammatory disease as a unique entity. J Pediatr. 1997 Apr;130(4):513-5.

Hoffman HM, Mueller JL, Broide DH, Wanderer AA, Kolodner RD. Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle-Wells syndrome. Nat Genet. 2001 Nov;29(3):301-5.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Kullenberg T, Löfqvist M, Leinonen M, Goldbach-Mansky R, Olivecrona H. Long-term safety profile of anakinra in patients with severe cryopyrin-associated periodic syndromes. Rheumatology (Oxford). 2016 Aug;55(8):1499-506. doi: 10.1093/rheumatology/kew208. Epub 2016 May 3.

Chang Z, Spong CY, Jesus AA, Davis MA, Plass N, Stone DL, Chapelle D, Hoffmann P, Kastner DL, Barron K, Goldbach-Mansky RT, Stratton P. Anakinra use during pregnancy in patients with cryopyrin-associated periodic syndromes (CAPS). Arthritis Rheumatol. 2014 Nov;66(11):3227-32.

Sibley CH, Plass N, Snow J, Wiggs EA, Brewer CC, King KA, Zalewski C, Kim HJ, Bishop R, Hill S, Paul SM, Kicker P, Phillips Z, Dolan JG, Widemann B, Jayaprakash N, Pucino F, Stone DL, Chapelle D, Snyder C, Butman JA, Wesley R, Goldbach-Mansky R. Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes. Arthritis Rheum. 2012 Jul;64(7):2375-86. doi: 10.1002/art.34409.

Goldbach-Mansky R, Dailey NJ, Canna SW, Gelabert A, Jones J, Rubin BI, Kim HJ, Brewer C, Zalewski C, Wiggs E, Hill S, Turner ML, Karp BI, Aksentijevich I, Pucino F, Penzak SR, Haverkamp MH, Stein L, Adams BS, Moore TL, Fuhlbrigge RC, Shaham B, Jarvis JN, O'Neil K, Vehe RK, Beitz LO, Gardner G, Hannan WP, Warren RW, Horn W, Cole JL, Paul SM, Hawkins PN, Pham TH, Snyder C, Wesley RA, Hoffmann SC, Holland SM, Butman JA, Kastner DL. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition. N Engl J Med. 2006 Aug 10;355(6):581-92.

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 )
Study First Received:	September 22, 2003
Results First Received:	May 6, 2016
Last Updated:	October 7, 2016

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