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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01576367
Recruitment Status : Completed
First Posted : April 12, 2012
Results First Posted : August 3, 2016
Last Update Posted : September 11, 2018
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Non-Randomized
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)
Actual Study Start Date : January 16, 2012
Actual Primary Completion Date : October 13, 2015
Actual Study Completion Date : October 13, 2015

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. [ Time Frame: Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months) ]
    Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result > 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity > minimal or Physician's Global Assessment >= minimal AND Skin Disease Assessment > minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe.

Secondary Outcome Measures :
  1. Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies [ Time Frame: minimum of 6 months and maximum of 24 months ]
    Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique.

  2. Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations [ Time Frame: Week 0, 80, 104, 128 and 152, last assessment ]
    CRP and SAA were used as serologic inflammatory markers. The target level concentrations for CRP and SAA was ≤15 mg/L and ≤10 mg/L, respectively. Negative change in concentration of inflammatory markers indicated improvement.

  3. Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease [ Time Frame: minimum of 6 months and maximum of 24 months ]
    Participants were assessed based by physician on Physician's Global Assessment measured on a 5--point scale for auto inflammatory disease activity as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe

  4. Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines [ Time Frame: pre-vaccine dose, Day 28 post-vaccine ]
    Participants who received any inactivated vaccines during the study were assessed for their ability to attain protective antibody levels against the vaccine (antigen) post immunization. Participants vaccinations were not assessed for a response if the antibody titre was already sufficient at pre-dose and maintained during the study.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 4 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01576367

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Novartis Investigative Site
Bruxelles, Belgium, 1200
Novartis Investigative Site
Laeken, Belgium, 1020
Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M5G 1X8
Novartis Investigative Site
Le Kremlin Bicetre, France, 94275
Novartis Investigative Site
Berlin, Germany, 13353
Novartis Investigative Site
Dresden, Germany, 01307
Novartis Investigative Site
Saint Augustin, Germany, 53757
Novartis Investigative Site
Tübingen, Germany, 72076
Novartis Investigative Site
Granada, Andalucia, Spain, 18012
Novartis Investigative Site
Valencia, Comunidad Valenciana, Spain, 46026
Novartis Investigative Site
Lausanne, Switzerland, 1011
United Kingdom
Novartis Investigative Site
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01576367    
Other Study ID Numbers: CACZ885D2307E1
2011-005154-57 ( EudraCT Number )
First Posted: April 12, 2012    Key Record Dates
Results First Posted: August 3, 2016
Last Update Posted: September 11, 2018
Last Verified: August 2018
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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
human monoclonal anti-human interleukin-1 antibody
autosomal dominant
familial autoinflammatory syndrome
childhood immunizations vaccinations
Additional relevant MeSH terms:
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Cryopyrin-Associated Periodic Syndromes
Pathologic Processes
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Skin Diseases, Infectious
Connective Tissue Diseases
Leukocyte Disorders
Hematologic Diseases