Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00069329
• Date of registration: 22/09/2003
• Prospective Registration: No
• Primary sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Public title: Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease
• Scientific title: A Long-Term Outcome Study With the IL-1 Receptor Antagonist Anakinra/Kineret in Patients With Neonatal Onset Multisystem Inflammatory Disease (NOMID/CINCA Syndrome) A Therapeutic Approach to Study the Pathogenesis of This Disease
• Date of first enrolment: September 2003
• Target sample size: 43
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00069329
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Raphaela T Goldbach-Mansky, M.D.
• Affiliation: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Key inclusion & exclusion criteria

-INCLUSION CRITERIA:

1. There is no age limitation.

2. Patients fulfill at least 2 of the following 3 clinical manifestations:

- Typical NOMID rash

- CNS involvement (papilledema, CSF pleocytosis, sensorineural hearing loss)

- Typical arthropathic changes on radiograph (epiphyseal and/or patellar
overgrowth.

3. Onset of manifestations of NOMID/CINCA at less than or equal to 6 months of age.

4. Stable dose of steroids, NSAIDs, DMARDs for 4 weeks prior to enrollment visit.

5. Washout period for biologics: 6 half-lives before anakinra administration for all
drugs with anti TNF properties. For etanercept (6 half-lives=24 days) this calculates
to drug discontinuation 3 days before enrollment into the observation period, for
infliximab and adalimumab (6 half-lives=48 days) drug will be discontinued 27 before
the observation period, and for thalidomide (6 half-lives=3 days) drug will be
discontinued for 3 days prior to anakinra administration.

6. Patient's or legal guardian's ability and willingness to give informed consent.

7. Females of childbearing potential (young women who have had at least one menstrual
period regardless of age) must have a negative urine pregnancy test at baseline prior
to performance of any radiologic procedure or administration of study medication.
Women of childbearing age and men able to father a child, who are sexually active,
will be asked to use a form of effective birth control, including abstinence.

8. Negative PPD test using 5 T.U. intradermal testing per CDC guidelines with exception
of inclusion criteria #9 below.

9. Patients with latent TB (positive PPD test) must have adequate therapy for TB
initiated prior to first dose of study medication as recommended in published
guidelines.

EXCLUSION CRITERIA:

1. Having received live virus vaccine during 3 months prior to baseline visit (1st visit
to NIH).

2. Patients with active infections or a history of pulmonary TB infection with or without
documented adequate therapy, Patients with current active TB, or recent close exposure
to an individual with active TB are excluded from the study.

3. Positive testing for HIV, Hepatitis B or C known or documented at screening,
enrollment or baseline visit.

4. Have a history of or concomitant diagnosis of congestive heart failure.

5. History of malignancy.

6. Recent use of IL-1 antagonist within the last three months or prior use of anti CD4
antibody.

7. Known hypersensitivity to E. coli derived products or any components of anakinra.

8. Presence of any other rheumatic disease or major chronic
infectious/inflammatory/immunologic disease (e.g. inflammatory bowel disease,
psoriatic arthritis, spondyloarthropathy, SLE in addition to NOMID/CINCA).

9. Presence of the following at enrollment visit: ALT or AST greater than 2.0 x upper
limit of normal (ULN) of the local laboratories values, creatinine greater than 1.5
xULN, WBC less than 3.6x10(9)/l; platelet count less than 150,000 mm(3).

10. Enrollment in any other investigational clinical study or receiving an investigational
agent, or has not yet completed at least 4 weeks since ending another investigational
device or drug trial.

11. Subjects for whom there is concern about compliance with the protocol procedures by
subject and/or parent/s and legally acceptable representative/s.

12. Lactating females or pregnant females.

13. Patients with asthma will only be included after evaluation by a pulmonary and
infectious disease consultation.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Papilledema

Urticaria

Arthropathy, Neurogenic

Nervous System Malformations

Intervention(s)

Drug: anakinra

Primary Outcome(s)

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Parent /Patient Pain Rating [Time Frame: Baseline]

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

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

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

Secondary ID(s)

030298

03-AR-0298

ZIAAR041138-08

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/12/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00069329