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 - Full Text View

Purpose

This trial will provide long-term safety, efficacy and tolerability of ACZ885 in Cryopyrin-associated Periodic Syndromes (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 Masking: Open Label 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:

Genetics Home Reference related topics: familial cold autoinflammatory syndrome familial Mediterranean fever Muckle-Wells syndrome neonatal onset multisystem inflammatory disease

Drug Information available for: Canakinumab

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]
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.

Secondary Outcome Measures:

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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: Yes ]
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 ] [ Designated as safety issue: Yes ]
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 ] [ Designated as safety issue: Yes ]
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 ] [ 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:	April 2015
Estimated Primary Completion Date:	November 2014 (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 Patients who did NOT experience sufficient symptomatic relief can receive a dose regimen adjustment. Other Name: Canakinumab

Eligibility

Ages Eligible for Study:	24 Months and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion criteria:

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).
Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.

Exclusion criteria:

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

Other protocol-defined inclusion/exclusion criteria may apply.

Contacts and Locations

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

Locations

Belgium
Novartis investigational Site	Recruiting
Laeken, Vilvoorde, Belgium, B-1800
Contact: Study Director 862-778-8300
Novartis Investigational Site	Recruiting
Bruxelles, Belgium
Contact: Study Director 862-778-8300
Germany
Novartis Investigational Site	Not yet recruiting
Dresden, Germany
Contact: Study Director 862-778-8300
Novartis Investigational Site	Not yet recruiting
Sankt Augustin, Germany
Contact: Study Director 862-778-8300
Novartis Investigational Site	Not yet recruiting
Tubingen, Germany
Contact: Study Director 862-778-8300

Sponsors and Collaborators

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:	April 10, 2012
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:

childhood immunizations/vaccinations.
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

Additional relevant MeSH terms:

Cryopyrin-Associated Periodic Syndromes
Cellulitis
Eosinophilia
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 May 19, 2013