Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01576367
First received: February 17, 2012
Last updated: August 7, 2014
Last verified: August 2014
  Purpose

This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study


Condition Intervention Phase
Cryopyrin-associated Periodic Syndromes
Familial Cold Autoinflammatory Syndrome
Muckle-Wells Syndrome
Neonatal Onset Multisystem Inflammatory Disease
Biological: ACZ885
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Primary Purpose: Treatment
Official Title: An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Assesment of the long-term efficacy of canakinumab with respect to the maintenance of treatment response in CAPS patients who completed the CACZ885D2307 study [ Time Frame: A minimum of 6 months and maximum of 24 months ] [ Designated as safety issue: No ]
    Response to treatment (maintained) and evidence of improvement will be collected through the Investigator's clinical assessment of autoinflammatory disease activity, clinical consultations and laboratory monitoring.


Secondary Outcome Measures:
  • Frequency of adverse events and the number of patients completing the extension study in the overall population [ Time Frame: minimum of 6 months and maximum of 24 months ] [ Designated as safety issue: Yes ]
    To measure safety and tolerability he occurrence of adverse events will be sought by non-directive questioning of the patient at each visit during the study. Adverse events also may be detected when they are volunteered by the patient during or between visits or through physical examination, laboratory test, or other assessments.

  • Change in inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA)) after treatment initiation [ Time Frame: minimum of 6 months and maximum of 24 months ] [ Designated as safety issue: Yes ]
    C-reactive protein (CRP) and serum amyloid A (SAA) will be measured at pre-specified timepoints during the study and at the time of dose adjustment.


Estimated Enrollment: 15
Study Start Date: January 2012
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: canakinumab
Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks)
Biological: ACZ885
Other Name: Canakinumab

  Eligibility

Ages Eligible for Study:   up to 4 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  1. Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
  2. Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
  3. Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.

Exclusion criteria:

  1. Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
  2. Patients who discontinued from the core CACZ885D2307 study

Other protocol-defined inclusion/exclusion criteria may apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01576367

Contacts
Contact: Novartis Pharmaceuticals +41613241111
Contact: Novartis Pharmaceuticals

Locations
Belgium
Novartis Investigative Site Completed
Bruxelles, Belgium, 1200
Novartis Investigative Site Completed
Laeken, Belgium, 1020
Canada, Ontario
Novartis Investigative Site Recruiting
Toronto, Ontario, Canada, M5G 1X8
France
Novartis Investigative Site Completed
Le Kremlin Bicetre, France, 94275
Novartis Investigative Site Not yet recruiting
Paris cedex 15, France, 75015
Germany
Novartis Investigative Site Active, not recruiting
Berlin, Germany, 13353
Novartis Investigative Site Recruiting
Dresden, Germany, 01307
Novartis Investigative Site Recruiting
St. Augustin, Germany, 53757
Novartis Investigative Site Recruiting
Tübingen, Germany, 72076
Spain
Novartis Investigative Site Recruiting
Granada, Andalucia, Spain, 18012
Novartis Investigative Site Recruiting
Valencia, Comunidad Valenciana, Spain, 46026
Switzerland
Novartis Investigative Site Not yet recruiting
Lausanne, Switzerland, 1011
United Kingdom
Novartis Investigative Site Recruiting
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01576367     History of Changes
Other Study ID Numbers: CACZ885D2307E1, 2011-005154-57
Study First Received: February 17, 2012
Last Updated: August 7, 2014
Health Authority: United States: Food and Drug Administration
Germany: Paul Ehrlich Institute
Spain: Spanish Agency of Medicines
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Belgium: Belgian Health Care Knowledge Center
Canada: Health Canada
Israel: Ministry of Health

Keywords provided by Novartis:
Cryopyrin-associated periodic syndromes (CAPS)
Familial Cold Autoinflammatory Syndrome (FCAS)
Muckle-Wells Syndrome (MWS)
Neonatal Onset Multisystem Inflammatory Disease (NOMID)
children, systemic autoinflammatory disease
CIAS-1 gene
NALP-3
NLRP3
ACZ885
canakinumab
human monoclonal anti-human interleukin-1 antibody
autosomal dominant
familial autoinflammatory syndrome
childhood immunizations vaccinations

Additional relevant MeSH terms:
Eosinophilia
Cryopyrin-Associated Periodic Syndromes
Cellulitis
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Skin Diseases, Infectious
Infection
Suppuration
Connective Tissue Diseases
Inflammation
Pathologic Processes
Leukocyte Disorders
Hematologic Diseases

ClinicalTrials.gov processed this record on August 21, 2014