View clinical trials related to CINCA Syndrome.
Filter by: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