Clinical trial 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
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cryopyrin-associated Periodic Syndromes
Familial Cold Autoinflammatory Syndrome
Muckle-Wells Syndrome
Neonatal Onset Multisystem Inflammatory Disease
Keywords: 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
childhood immunizations vaccinations
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-04-12
Enrollment: 17
Sex: ALL
Minimum age (years): 1
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
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.
timeFrame: Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)
Secondary outcomes:
measure: Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies
timeFrame: minimum of 6 months and maximum of 24 months
measure: Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations
timeFrame: Week 0, 80, 104, 128 and 152, last assessment
measure: Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease
timeFrame: minimum of 6 months and maximum of 24 months
measure: Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines
timeFrame: pre-vaccine dose, Day 28 post-vaccine
Other outcomes:
Locations:
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Laeken
country: Belgium
facility: Novartis Investigative Site
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Le Kremlin Bicetre
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Saint Augustin
country: Germany
facility: Novartis Investigative Site
city: Tübingen
country: Germany
facility: Novartis Investigative Site
city: Granada
country: Spain
facility: Novartis Investigative Site
state: Andalucia
city: Valencia
country: Spain
facility: Novartis Investigative Site
state: Comunidad Valenciana
city: Lausanne
country: Switzerland
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Brogan PA, Hofer M, Kuemmerle-Deschner JB, Kone-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penades I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9.