Long‐Term Efficacy and Safety of Canakinumab in Patients With Tumor Necrosis Factor Receptor–Associated Periodic Syndrome: Results From a Phase III Trial

Abstract

ObjectiveWe aimed at assessing efficacy, safety, and tolerability of canakinumab in patients with tumor necrosis factor receptor–associated periodic syndrome (TRAPS) during a 72‐week long‐term, open‐label extension of the CLUSTER study.MethodsPatients received open‐label canakinumab 150 or 300 mg, either every 4 weeks (q4w) or every 8 weeks, with up‐titration permitted after on‐treatment flares (maximum dose: 300 mg q4w). Efficacy assessments included physician global assessment of disease activity, number of flares, and serum C‐reactive protein (CRP) and serum amyloid A protein (SAA) levels. Adverse events were also reported. Results are described for the overall population and according to the cumulative dose of canakinumab adjusted for body weight (<36 mg/kg or ≥36 mg/kg).ResultsOf 53 patients entering the final phase (epoch 4) of CLUSTER, 51 completed the treatment. At the end of epoch 4, >94% of patients achieved no or minimal disease activity. Most patients had either no (69.8%) or one flare (24.5%), whereas at baseline, the median number of flares was 9.0 per year. Median CRP levels remained at <10 mg/L. Median SAA concentrations were largely unchanged, with medians of 11.5 mg/L and 14.5 mg/L in the <36 mg/kg and ≥36 mg/kg groups, respectively, at the end of the study. No unexpected safety findings were identified.ConclusionControl of disease activity, with low flare incidence, was maintained with long‐term canakinumab treatment in patients with TRAPS during the 72‐week final epoch of the CLUSTER study, with no new safety findings.