Clinical trial NCT01105507: The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cryopyrin Associated Periodic Syndrome
Keywords: 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)
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-04-16
Enrollment: 4
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Male and female patients at least 4 years of age at the time of the screening visit
2. Patient's informed consent for \> or = 18 years of age before any assessment is performed
3. 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.
4. Patients with a diagnosis of CAPS: FCAS/FCU, MWS, or NOMID/CINCA
5. Body weight \> or = 15 kg
6. Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary)

Exclusion Criteria:

1. Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at baseline
2. 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.
3. Participation in any clinical investigation within 4 weeks prior to dosing
4. Live vaccinations within 3 months prior to the start of the trial, during the trial and up to 3 months following the last dose.
5. 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).
6. History of drug or alcohol abuse within 12 months prior to dosing
7. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes

"Other protocol-defined inclusion/exclusion criteria may apply"

Primary outcomes:
measure: 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.
timeFrame: 18 months
Secondary outcomes:
measure: Change in long term maintenance of Health-Related Quality of Life and productivity measured from baseline to end of study.
timeFrame: Baseline, 18 months
measure: Treatment adherence with canakinumab for the duration of the study.
timeFrame: 18 months
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Alberta Children's Hospital, Department of Pediatrics
city: Halifax
country: Canada
facility: Queen Elizabeth II Hospital
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: