To date there are no approved effective therapies for the treatment of cryopyrin-associated periodic syndromes (CAPS) including Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), or Neonatal Onset Multisystem Inflammatory Disease (NOMID) in Japan.
The study will assess the efficacy and safety of canakinumab in Japanese patients with cryopyrin-associated periodic syndromes (CAPS). In previous and currently ongoing CAPS studies (CACZ885A2102, CACZ885D2201, CACZ885D2304, CACZ885D2306), it has been observed that treatment with canakinumab in patients with CAPS contributed to ensure absence of relapse, to improve signs and symptoms and to prevent secondary disease complications. However, no Japanese patients have been included in those studies. This study will allow access for Japanese patients to a new potentially efficacious treatment for CAPS patients with a convenient dosing regimen.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Cryopyrin-associated Periodic Syndromes
canakinumab
Novartis Investigative site
Fukuoka
Japan
Active, not recruiting
Published on BioPortfolio: 2014-07-23T21:12:34-0400
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months.
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Cryopyrin-associated Periodic Syndromes
A group of rare autosomal dominant diseases, commonly characterized by atypical URTICARIA (hives) with systemic symptoms that develop into end-organ damage. The atypical hives do not involve T-cell or autoantibody. Cryopyrin-associated periodic syndrome includes three previously distinct disorders: Familial cold autoinflammatory syndrome; Muckle-Wells Syndrome; and CINCA Syndrome, that are now considered to represent a disease continuum, all caused by NLRP3 protein mutations.
Aortic Arch Syndromes
Conditions resulting from abnormalities in the arteries branching from the ASCENDING AORTA, the curved portion of the aorta. These syndromes are results of occlusion or abnormal blood flow to the head-neck or arm region leading to neurological defects and weakness in an arm. These syndromes are associated with vascular malformations; ATHEROSCLEROSIS; TRAUMA; and blood clots.
Periodic Acid-schiff Reaction
A histochemical technique for staining carbohydrates. It is based on PERIODIC ACID oxidation of a substance containing adjacent hydroxyl groups. The resulting aldehydes react with Schiff reagent to form a colored product.
Malabsorption Syndromes
General term for a group of MALNUTRITION syndromes caused by failure of normal INTESTINAL ABSORPTION of nutrients.
Immunologic Deficiency Syndromes
Syndromes in which there is a deficiency or defect in the mechanisms of immunity, either cellular or humoral.