A 24-month open-label study of canakinumab in neonatal-onset multisystem inflammatory disease.

ABSTRACT:

Objective

To study efficacy and safety of escalating doses of canakinumab, a fully human anti-IL-1β monoclonal antibody in the severe cryopyrin-associated periodic syndrome, neonatal-onset multisystem inflammatory disease (NOMID).

Methods

6 patients were enrolled in this 24-month, open-label phase I/II study. All underwent anakinra withdrawal. The initial subcutaneous canakinumab dose was 150 mg (or 2 mg/kg in patients ≤40 kg) or 300 mg (or 4 mg/kg) with escalation up to 600 mg (or 8 mg/kg) every 4 weeks. Full remission was remission of patient-reported clinical components and measures of systemic inflammation and CNS inflammation. Hearing, vision and safety were assessed. Primary endpoint was full remission at month 6.

Results

All patients flared after anakinra withd

SUBMITTER: Sibley CH 

PROVIDER: S-EPMC4258169 | biostudies-literature | 2015 Sep

REPOSITORIES: biostudies-literature