Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome
About this trial
This is an interventional treatment trial for Cryopyrin-associated Periodic Syndromes focused on measuring Cryopyrin-associated periodic syndromes (CAPS), Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), Neonatal Onset Multisystem Inflammatory Disease (NOMID), children, systemic autoinflammatory disease, CIAS-1 gene, NALP-3, NLRP3, ACZ885, canakinumab, human monoclonal anti-human interleukin-1 antibody, autosomal dominant, familial autoinflammatory syndrome, childhood immunizations vaccinations
Eligibility Criteria
Inclusion criteria:
- Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
- Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
- Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.
Exclusion criteria:
- Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
- Patients who discontinued from the core CACZ885D2307 study
Other protocol-defined inclusion/exclusion criteria may apply.
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Experimental
canakinumab
Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks)