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

NCT01105507 | Completed Phase 3

• 1 other identifier: CACZ885DCA01
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

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.

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)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• 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

Study Arms (1)

canakinumab arm

EXPERIMENTAL

Drug: canakinumab (company code: ACZ885D)

Interventions

canakinumab (company code: ACZ885D) DRUG

canakinumab arm

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (2)

Alberta Children's Hospital, Department of Pediatrics

Calgary, Canada

Location

Queen Elizabeth II Hospital

Halifax, Canada

Location

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2010
• First Posted: April 16, 2010
• Study Start: August 1, 2010
• Primary Completion: May 12, 2012
• Study Completion: May 12, 2012
• Last Updated: March 21, 2017

Record last verified: 2017-03

Locations

CA (2)