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Completed

NCT Number: NCT01576367

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

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Key information

Conditions

Cryopyrin-associated Periodic Syndromes Chronic Disease Chronic Inducible Urticaria Chronic Urticaria Cold Urticaria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Familial Cold Autoinflammatory Syndrome Genetic Diseases, Inborn Hereditary Autoinflammatory Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Muckle-Wells Syndrome Multiple Pterygium Syndrome, Autosomal Dominant Neonatal Onset Multisystem Inflammatory Disease Pathologic Processes Pathological Conditions, Signs and Symptoms Skin Diseases Skin Diseases, Genetic Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria

Age range

1 year–4 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novartis Investigative Site, Brussels, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ACZ885

Biological

Other names: Canakinumab

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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)

Important dates

Study start
2012
Primary completion
2015
Study completion
2015
First posted
Apr 12, 2012
Registry last updated
Sep 11, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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