DDrare: Database of Drug Development for Rare Diseases

Searched query = "Cryopyrin-associated periodic syndrome", "Cryopyrin associated periodic fever syndrome", "Familial cold autoinflammatory syndrome", "FCAS", "Mucke-Wells syndrome", "MWS", "Chronic infantile neurologic cutaneous, and articular syndrome", "CINCA syndrome", "Neonatal onset multisystem inflammatory disease", "NOMID"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ACZ885; ACZ885 drug substance; ATI-450; Anakinra; Anti-interleukin-1B monoclonal antibody; C-028380; CANAKINUMAB; Canakinumab; Canakinumab (ACZ885); Canakinumab (WHO approval pending); Canakinumab (company code: ACZ885D); ILARIS; IZD174; IZD334; Ilaris; Inzomelid; Kineret; Recombinant human monoclonal antibody to human IL-1Beta of the IgG1/K class; Recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class; Rilonacept; Rilonacept 160 mg; Tranilast; XOMA 052

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2004-002980-26-FR (EUCTR)	07/06/2007	29/03/2007	An open-label, phase II dose titration study of ACZ885 (human anti-IL-1beta monoclonal antibody) to assess the clinical efficacy, safety, pharmacokinetics and pharmacodynamics in patients with NALP3 mutations - A2102	An open-label, phase II dose titration study of ACZ885 (human anti-IL-1beta monoclonal antibody) to assess the clinical efficacy, safety, pharmacokinetics and pharmacodynamics in patients with NALP3 mutations - A2102	Muckle-Wells Syndrome: rare hereditary, autosomal dominant, systemic inflammatory disease, characterized by recurrent episodes of fever, arthralgia, myalgia, urticarial rash, and conjunctivitis. Laboratory findings show an elevation of acute phase proteins such as CRP and SAA, a high ESR, together with leukocytosis, and hypergammaglobulinemia. Severe long term complications include progressive sensorineural deafness, and systemic AA amyloidosis. MedDRA version: 9.1;Level: LLT;Classification code 10064573;Term: Neonatal-onset multisystemic inflammatory disease MedDRA version: 9.1;Classification code 10064570;Term: Familial cold autoinflammatory syndrome	Product Code: ACZ885 Other descriptive name: ACZ885 drug substance	Novartis Pharma Services AG	NULL	Not Recruiting			Female: yes Male: yes	34	Phase 2	France;Spain;Germany;United Kingdom

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EUCTR2004-002980-26-FR
(EUCTR)

07/06/2007

29/03/2007

An open-label, phase II dose titration study of ACZ885 (human anti-IL-1beta monoclonal antibody) to assess the clinical efficacy, safety, pharmacokinetics and pharmacodynamics in patients with NALP3 mutations - A2102

Muckle-Wells Syndrome: rare hereditary, autosomal dominant, systemic inflammatory disease, characterized by recurrent episodes of fever, arthralgia, myalgia, urticarial rash, and conjunctivitis. Laboratory findings show an elevation of acute phase proteins such as CRP and SAA, a high ESR, together with leukocytosis, and hypergammaglobulinemia. Severe long term complications include progressive sensorineural deafness, and systemic AA amyloidosis.
MedDRA version: 9.1;Level: LLT;Classification code 10064573;Term: Neonatal-onset multisystemic inflammatory disease
MedDRA version: 9.1;Classification code 10064570;Term: Familial cold autoinflammatory syndrome

Product Code: ACZ885
Other descriptive name: ACZ885 drug substance

Novartis Pharma Services AG

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

France;Spain;Germany;United Kingdom