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

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

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.

Study Overview

• Status: Completed
• Conditions: Cryopyrin Associated Periodic Syndrome
• Intervention / Treatment: Drug: canakinumab (company code: ACZ885D)

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Calgary, Canada
    • Alberta Children's Hospital, Department of Pediatrics
  • Halifax, Canada
    • Queen Elizabeth II Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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"

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: canakinumab arm	Drug: canakinumab (company code: ACZ885D)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (Actual): May 12, 2012
• Study Completion (Actual): May 12, 2012

Study Registration Dates

• First Submitted: March 25, 2010
• First Submitted That Met QC Criteria: April 15, 2010
• First Posted (Estimate): April 16, 2010

Study Record Updates

• Last Update Posted (Actual): March 21, 2017
• Last Update Submitted That Met QC Criteria: March 17, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Keywords: Cutaneous; Canakinumab; Familial Cold Autoinflammatory Syndrome (FCAS); Muckle-Wells Syndrome (MWS); Cryopyrin-associated periodic syndromes; Familial Cold Urticaria (FCU); Neonatal-Onset Multisystem Inflammatory Disease (NOMID); Chronic Infantile Neurological; Articular Syndrome (CINCA); Treatment of patients diagnosed with CAPS (Cryopyrin associated periodic syndrome)
• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Disease; Genetic Diseases, Inborn; Hereditary Autoinflammatory Diseases; Skin Diseases, Genetic; Syndrome; Cryopyrin-Associated Periodic Syndromes
• Other Study ID Numbers: CACZ885DCA01