ClinicalTrials.gov
ClinicalTrials.gov Menu

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.
ClinicalTrials.gov Identifier: NCT01576367
Recruitment Status : Completed
First Posted : April 12, 2012
Results First Posted : August 3, 2016
Last Update Posted : September 11, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

February 17, 2012
April 12, 2012
June 22, 2016
August 3, 2016
September 11, 2018
January 16, 2012
October 13, 2015   (Final data collection date for primary outcome measure)
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.
Long-term efficacy of canakinumab with respect to the maintenance of treatment response in CAPS patients who completed the CACZ885D2307 study [ Time Frame: minimum of 6 months and maximum of 24 months ]

Outcome Measure Description:

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.

Complete list of historical versions of study NCT01576367 on ClinicalTrials.gov Archive Site
  • 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.
  • 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.
  • 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
  • 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.
  • Safety and tolerability as assessed by the overall 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 ]

    The 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.

    Safety Issue:

  • The presence of protective antibody levels following immunization with inactivated (killed) vaccines administered during the extension study [ Time Frame: minimum of 6 months and maximum of 24 months ]
    Assessment of protective antibody titers (IgG) against the following antigens are performed: Diphteria, Pertussis, Tetanus, Haemophilus influenzae type b, Influenza, Pneumococcus, Meningococcus, Hepatitis A & Hepatitis B
  • Safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination during the extension study [ Time Frame: minimum of 6 months and maximum of 24 months ]
    The occurrence of adverse events or reactions 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.
  • The number of patients who relapse during the extension study as determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers [ Time Frame: minimum of 6 months and maximum of 24 months ]
    Relapse will be defined according to the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers
  • Inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA)) after treatment initiation [ Time Frame: A minimum of 6 months and maximum of 24 months ]
    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.
Not Provided
Not Provided
 
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
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)
This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Primary Purpose: Treatment
  • Cryopyrin-associated Periodic Syndromes
  • Familial Cold Autoinflammatory Syndrome
  • Muckle-Wells Syndrome
  • Neonatal Onset Multisystem Inflammatory Disease
Biological: ACZ885
Other Name: Canakinumab
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)
Intervention: Biological: ACZ885
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
17
15
October 13, 2015
October 13, 2015   (Final data collection date for primary outcome measure)

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.

Sexes Eligible for Study: All
1 Year to 4 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Belgium,   Canada,   France,   Germany,   Spain,   Switzerland,   United Kingdom
 
 
NCT01576367
CACZ885D2307E1
2011-005154-57 ( EudraCT Number )
Yes
Not Provided
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP