The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada

Study Overview
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Brief Summary
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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.

Official Title
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An Open Label Study to Assess the Safety, Tolerability and Efficacy of Canakinumab (ACZ885) in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada

Conditions
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Cryopyrin Associated Periodic Syndrome

Intervention / Treatment
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* Drug: canakinumab (company code: ACZ885D)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients at least 4 years of age at the time of the screening visit Patient's informed consent for > or = 18 years of age before any assessment is performed Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 18 years of age. Patients with a diagnosis of CAPS: FCAS/FCU, MWS, or NOMID/CINCA Body weight > or = 15 kg Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary) Exclusion Criteria: Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at baseline Patient of childbearing potential (man or woman) and unwilling to use double barrier method of contraception. It is required that double barrier method of contraception be used (i.e. condom with spermicide or diaphragm with spermicide) by patients of childbearing potential (man or woman) regardless of whether a hormonal agent is also used as a method of contraception. Participation in any clinical investigation within 4 weeks prior to dosing Live vaccinations within 3 months prior to the start of the trial, during the trial and up to 3 months following the last dose. History of significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty). History of drug or alcohol abuse within 12 months prior to dosing History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes Other protocol-defined inclusion/exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 4 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: canakinumab arm<br> | Drug: canakinumab (company code: ACZ885D)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety & tolerability of canakinumab by measuring the maintenance of response over time using the Investigators Clinical Assessment of Disease Activity scale and the evaluation of serum inflammation markers CRP (C-Reactive Protein), SAA (Serum Amyloid A. |  | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in long term maintenance of Health-Related Quality of Life and productivity measured from baseline to end of study. |  | Baseline, 18 months | 
| Treatment adherence with canakinumab for the duration of the study. |  | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Canakinumab, Cryopyrin-associated periodic syndromes, Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria (FCU), Muckle-Wells Syndrome (MWS), Neonatal-Onset Multisystem Inflammatory Disease (NOMID), Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA), Treatment of patients diagnosed with CAPS (Cryopyrin associated periodic syndrome)