Biological Treatments: New Weapons in the Management of Monogenic Autoinflammatory Disorders

Table 2

Recent and ongoing clinical trials on biologic treatments in the monogenic autoinflammatory disorders.

	Phase	Status	Study	Disease	ClinicalTrials.gov identifier
Anakinra	III	Not yet recruiting	Kineret (Anakinra) in adult patients with colchicine-resistant familial Mediterranean fever	FMF	NCT01705756
	I	Completed	The use of kineret (anakinra) in the treatment of familial cold urticaria	FCAS	NCT00214851
	II	Recruiting	Anakinra to treat patients with neonatal onset multisystem inflammatory disease	NOMID	NCT00069329
	II	Recruiting	Anakinra for inflammatory pustular skin diseases	PAPAs	NCT01794117
	III	Terminated	Canakinumab to treat neonatal-onset multisystem inflammatory disease	NOMID	NCT00770601
	III	Recruiting	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	CAPS	NCT01576367
	III	Completed	The safety and efficacy of canakinumab in patients aged 4 years or older diagnosed with cryopyrin-associated periodic syndromes (CAPS) in Canada	CAPS	NCT01105507
	III	Completed	Efficacy and safety study of canakinumab administered for 6 months (24 weeks) in japanese patients with cryopyrin-associated periodic syndromes followed by an extension phase	CAPS	NCT00991146
	II	Active, not recruiting	Evaluation of the safety and efficacy of canakinumab in pediatric patients with colchicine intolerant or colchicine-resistant familial Mediterranean fever (FMF) (CONTROL FMF)	FMF	NCT01148797
	II	Completed	Efficacy and safety, of canakinumab in patients with colchicine-resistant familial Mediterranean fever	FMF	NCT01088880
Canakinumab	III	Recruiting	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	CAPS	NCT01302860
	III	Completed; Has results	Efficacy and safety of ACZ885 in patients with the following cryopyrin-associated periodic syndromes: familial cold autoinflammatory syndrome, Muckle-Wells syndrome, or neonatal onset multisystem inflammatory disease	CAPS	NCT00685373
	III	Terminated	Canakinumab to treat neonatal-onset multisystem inflammatory disease	NOMID	NCT00770601
	II	Recruiting	Canakinumab for pyoderma gangrenosum		NCT01302795
	—	Recruiting	Clinical outcomes and safety: a registry study of ilaris (canakinumab) patients (B-confident)	CAPS	NCT01213641
	II	Recruiting	Canakinumab in patients with active hyper-IgD syndrome	MKD	NCT01303380
	II	Completed	Safety, efficacy, pharmacokinetics, and pharmacodynamics of ACZ885 in patients with NALP3 mutations and clinical symptoms	CAPS	NCT00487708
	III	Completed; Has results	Efficacy, safety, and tolerability of ACZ885 in patients with Muckle-Wells syndrome (REMITTER)	MWS	NCT00465985
	II	Active, not recruiting	Efficacy and safety study of ACZ885 in patients with active recurrent or chronic (Tumor Necrosis Factor) TNF-receptor-associated periodic syndrome (TRAPS)	TRAPS	NCT01242813
Rilonacept	II	Completed; Has results	Rilonacept for treatment of familial Mediterranean fever (FMF)	FMF	NCT00582907
	III	Completed; Has results	Rilonacept for treatment of cryopyrin-associated periodic syndromes (CAPS)	CAPS	NCT00288704
	II	Completed	Safety and tolerability of rilonacept in Muckle-Wells syndrome (MWs) or Schnitzler syndrome (ACCILTRA1)	MWS	NCT01045772
	II	Recruiting	Rilonacept for deficiency of the interleukin-1 receptor antagonist (DIRA)	DIRA	NCT01801449
	II	Completed	Interleukin-1 trap to treat autoinflammatory disease	FMF, CAPS	NCT00094900

FMF: familial Mediterranean fever; TRAPS: tumor necrosis factor receptor-associated periodic syndrome; FCAS: familial cold autoinflammatory syndrome; MWS: Muckle-Wells syndrome; NOMID: neonatal onset multisystem inflammatory disease; MKD: mevalonate kinase deficiency syndrome; PAPAs: pyogenic arthritis, pyoderma gangrenosum, acne syndrome; DIRA: deficiency of interleukin-1 receptor antagonist.