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

Novartis Pharmaceuticals17 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Enrollment

Conditions

Cryopyrin-associated Periodic Syndromes Familial Cold Autoinflammatory Syndrome Muckle-Wells Syndrome Neonatal Onset Multisystem Inflammatory Disease

Interventions

Eligibility

Sex: ALLMin age: 1 YearMax age: 4 Years

Medical Language ↔ Plain English

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.

Locations (12)

Novartis Investigative Site

Brussels, Belgium

Novartis Investigative Site

Laken, Belgium

Novartis Investigative Site

Toronto, Ontario, Canada

Novartis Investigative Site

Le Kremlin-Bicêtre, France

Novartis Investigative Site

Berlin, Germany

Novartis Investigative Site

Dresden, Germany

Novartis Investigative Site

Saint Augustin, Germany

Novartis Investigative Site

Tübingen, Germany

Novartis Investigative Site

Granada, Andalusia, Spain

Novartis Investigative Site

Valencia, Valencia, Spain

...and 2 more locations

Outcomes

Primary Outcomes

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.

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.

Time frame: Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)

Secondary Outcomes

Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies

Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique.

Time frame: minimum of 6 months and maximum of 24 months

Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations

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.

Time frame: Week 0, 80, 104, 128 and 152, last assessment

Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease

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

Time frame: minimum of 6 months and maximum of 24 months

Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines

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.

Time frame: pre-vaccine dose, Day 28 post-vaccine