Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease

Stopped early · Phase 3

Conditions studied: NOMID, CINCA Syndrome

In brief

This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous, articular (CINCA) syndrome. This disease can cause rash, joint deformities, brain inflammation, problems with the eyes and learning difficulties. Canakinumab is an experimental drug that inhibits the action of a protein produced by the body called human IL-1beta, which is responsible for the symptoms in NOMID and also contributes to many other kinds of inflammatory diseases. Patients 2 years of age and older with NOMID / CINCA may be eligible for this study. Participants undergo the following procedures: Screening Phase * Medical history and review of medical records * Blood tests * Daily diary of symptoms and medicines take Washout/Lead-in Phase * Discontinuation of anakinra or other medications, a 6 to 48-hour run-in period (only for patients who discontinued anakinra or other IL-1 blocking therapy). Treatment Phase * Injection of canakinumab under the skin every 8 weeks for 6 months * Monitoring and evaluations during treatment, including: * Quality-of-life questionnaires and daily diary * Vital signs measurements (heartbeat, blood pressure, temperature) * Blood tests * Electrocardiogram * Tuberculosis skin test * Neurological, eye and skin examinations at beginning and end of study * Cognitive evaluation at beginning and end of study * Lumbar puncture (spinal tap) at the beginning of the study, 2 weeks after the second dosing of canakinumab and at the end of the study * X-rays and bone density scan at beginning and end of study * Magnetic resonance imaging (MRI) of the head at beginning and end of study Follow-up Phase * Monthly clinic visits after the last dose of canakinumab for a minimum of 60 days End-of-Study Evaluation * Series of tests 8 weeks after last dose of canakinumab to evaluate treatment response and side effects

Key facts

Study ID

NCT00770601

Run by

Novartis Pharmaceuticals

People needed

7

Starts

2009-01-26

Expected to finish

2011-02-17

Last updated by the study team

2021-06-10

Who can join

Age: 2 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Subjects meeting any of the following criteria will be excluded from entry into or continuation in the study unless sponsor approval is obtained:
• Pregnant or breastfeeding women.
• Participation in any clinical trial investigation within 4 weeks prior to dosing or longer if required by local regulation, with the exception of trials with anakinra and/or canakinumab.
• In case of previous treatment with biologic agents or DMARDs, an appropriate washout period (as according to the recognized duration of effect and half lives) will be required for such patients to be eligible to participate in the trial, e.g.:
• Previous treatment and required washout period prior to baseline and thereafter:
• Rituximab, 26 weeks
• Infliximab, 12 weeks
• Adalimumab, 8 weeks
• Etanercept, 4 weeks
• Anakinra, 1 day
• Any other investigational biologics, 8 weeks
• Leflunomide, 4 weeks
• Thalidomide, 4 weeks
• Cyclosporine, 4 weeks
• i.v. immunoglobulin (i.v. Ig), 8 weeks
• Dapsone, mycophenolate mofetil, 3 weeks
• Corticosteroids greater than or equal to 20 mg/day or greater than 0.4 mg/kg prednisone or prednisone equivalent, whichever applies, 1 week
• Donation or loss of 300 mL or more of blood within 8 weeks prior to dosing.
• A past medical history of clinically significant ECG abnormalities or a family history of a prolonged QT-interval syndrome.
• History of immunocompromise.
• Positive test for or prior history of HIV (ELISA and Western blot), Hepatitis B (Hepatitis B surface antigen) or Hepatitis C.
• Presence of active infections or a history of pulmonary TB infection with or without documented adequate therapy. Subjects with current active TB, or recent close exposure to an individual with active TB are excluded from the study.
• Presence of any additional rheumatic disease or significant systemic disease. For example, major chronic infectious/ inflammatory/ immunologic disease (such as inflammatory bowel disease, psoriatic arthritis, spondyloarthropathy, systemic lupus erythematosus in addition to the autoinflammatory disease).
• Treatment with a live virus vaccine during 3 months prior to baseline visit. No live vaccines will be allowed throughout the course of this study and up to 3 months following the last dose.
• History of renal transplant.

Where it is running

• National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), 9 — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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