Table 4.

Principal clinical trials involving IL-1 antagonists for CAPS patients.

Author, year/reference number	Study type, NCT number	Drug(s)	Details of treatment	Number of patients, clinical phenotype	Study duration	Main study results
Goldbach-Mansky et al., 2006 (126)	Open label, NCT00069329	Anakinra	1-to-2- mg/kg/day	18, CINCA/NOMID	6 months	Clinical remission in all patients Reduction of SAA, CRP, ESR serum level Improvement of CNS abnormalities, shown by MRI
Sibley et al., 2012 (121)	Open label, NCT00069329	Anakinra	1-to-5 mg/kg/day	26, CINCA/NOMID	60 months	Improvement in patients’ and physicians’ disease scores Reduction of systemic inflammatory markers Significant decrease in CNS inflammation
Goldbach-Mansky et al., 2008 (120)	Open label, NCT00094900	Rilonacept	300 mg (induction dose) 100-to-360 mg/week (maintenance dose)	5, FCAS	24 months	Clinical remission and decrease in inflammatory markers
Hoffman et al.,2012 (73)	Open label, NCT00288704	Rilonacept	160 mg/week (adults) 2.2 mg/kg (paediatrics)	101, - 95 FCAS - 3 MWS - 3 FCAS/MWS overlap	18 months	Improvement of clinical sign and symptoms, and normalized inflammatory markers
Lachmann et al., 2009 (76)	RCT, NCT00465985	Canakinumab, placebo	150 mg every 8 weeks	35, - 34 MWS - 1 MWS/CINCA/NOMID overlap	6 months	90% clinical remission in treatment group Normal levels of SAA, CRP in treatment group
Kuemmerle-Deschner et al., 2011 (131)	Open label, NCT00685373	Canakinumab,	150 mg every 8 weeks	166, - 30 FCAS - 103 MWS - 32 CINCA/NOMID	24 months	Complete remission in 78% canakinumab-naive patients Improvement in clinical and laboratory parameters in the entire cohort

CINCA/NOMID, Chronic infantile neurologic, cutaneous, and articular syndrome/neonatal-onset multisystem inflammatory disease; SAA, serum amyloid A protein; CRP, C-reactive protein; ESR, erythrocyte sedimentation rate; CNS, central nervous system; MWS, Muckle Wells Syndrome; FCAS, Familial cold autoinflammatory syndrome; RCT, Randomized controlled study.