Clinical Trial: Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional




Official Title: A Multi-Center, Open Label, 24-Month Treatment Study to Establish the Safety, Tolerability, Efficacy, Pharmacokinetics of Canakinumab (Anti-IL-1 Beta Antibody) in Patients With NOMID / 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 interleukin 1, 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 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

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 te