Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease
Status:
Terminated
Trial end date:
2011-02-17
Target enrollment:
Participant gender:
Summary
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
Phase:
Phase 3
Details
Lead Sponsor:
Novartis Pharmaceuticals
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)