Long-term efficacy/safety of canakinumab in patients with tumor necrosis factor receptor-associated periodic syndrome: results from a phase III trial.

OBJECTIVES: To assess 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.

METHODS: Patients received open-label canakinumab 150 or 300 mg, either every 4 weeks (q4w) or every 8 weeks, with up-titration permitted following on-treatment flares (maximum dose: 300 mg q4w). Efficacy assessments included physician global assessment (PGA) 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).

RESULTS: Of 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 study. No unexpected safety findings were identified.

CONCLUSION: Control 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. This article is protected by copyright. All rights reserved.