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

Cryopyrin-associated Periodic Syndromes Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01576367
• Other study ID #: CACZ885D2307E1
• Secondary ID: 2011-005154-57
• Status: Completed
• Phase: Phase 3
• Start date: January 16, 2012
• Est. completion date: October 13, 2015

Study information

• Verified date: August 2018
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: October 13, 2015
• Est. primary completion date: October 13, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 4 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cellulitis
• Cryopyrin-Associated Periodic Syndromes
• Eosinophilia
• Familial Cold Autoinflammatory Syndrome
• Muckle-Wells Syndrome
• Neonatal Onset Multisystem Inflammatory Disease
• Syndrome

Intervention

Biological:
• ACZ885

Locations

Country	Name	City	State
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Laeken
Canada	Novartis Investigative Site	Toronto	Ontario
France	Novartis Investigative Site	Le Kremlin Bicetre
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Dresden
Germany	Novartis Investigative Site	Saint Augustin
Germany	Novartis Investigative Site	Tübingen
Spain	Novartis Investigative Site	Granada	Andalucia
Spain	Novartis Investigative Site	Valencia	Comunidad Valenciana
Switzerland	Novartis Investigative Site	Lausanne
United Kingdom	Novartis Investigative Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Belgium,  Canada,  France,  Germany,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)
Secondary	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.	minimum of 6 months and maximum of 24 months
Secondary	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.	Week 0, 80, 104, 128 and 152, last assessment
Secondary	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	minimum of 6 months and maximum of 24 months
Secondary	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.	pre-vaccine dose, Day 28 post-vaccine