Clinical trial NCT01213641: Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients
Official title: An Open-label, Long-term, Prospective, Observational Study to Monitor the Safety and Effectiveness of Ilaris in CAPS Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cryopyrin-associated Periodic Syndromes (CAPS)
Familial Cold Autoinflam Syn (FCAS)
Muckle-wells Syn (MWS)
Neonatal Onset Multisystem Inflam Disease (NOMID)
Keywords: Cryopyrin-associated periodic syndromes(CAPS)
CIAS1 protein
NLRP3 protein
human cryopyrin protein
Familial Cold Autoinflammatory Syndrome(FCAS)
Muckle-Wells Syndrome(MWS)
Neonatal Onset Multisystem Inflammatory disease(NOMID)
NALP3
canakinumab
Interleukin-1(IL-1)
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-10-04
Enrollment: 288
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with CAPS and treated with canakinumab. Patients treated with canakinumab for other autoinflammatory diseases may be included where allowed per local regulations.

Brief summary: The purpose of this observational study is to collect additional information regarding long-term safety and effectiveness of Ilaris in the treatment of CAPS patients in clinical practice.


Eligibility criteria: Inclusion Criteria:

* Patients receiving Ilaris (canakinumab) at the time of enrollment as part of medical care

Exclusion Criteria:

* Local regulations in some locations may exclude patients who receive Ilaris for a non-approved indication
* Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: To monitor and further explore the overall safey of canakinumab focusing in serious infections
timeFrame: At least 5 years
Secondary outcomes:
measure: Long-term impact of Ilaris on disease progression (including systemic AA amyloidosis as evidenced by renal function, neurologic and ophthalmologic symptoms, and sensorineural deafness)
timeFrame: At least 5 years
measure: Growth and development patterns of children aged 4 to 18 years of age exposed to Ilaris
timeFrame: at least 5 years
measure: Identify previously unrecognized serious adverse drug reactions in the treated population
timeFrame: at least 5 years
measure: Usage and patterns of dosing of Ilaris in routine clinical practice
timeFrame: at least 5 years
measure: Incidence of serious infections
timeFrame: at least 5 years
measure: Incidence of malignancies
timeFrame: at least 5 years
measure: Incidence of hypersensitivity reactions
timeFrame: at least 5 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Little Rock Allergy and Asthma Clinic
state: Arizona
city: Columbus
country: United States
facility: Allergy Center at Brookstone
state: Georgia
city: Chicago
country: United States
facility: Rush Presbyterian - St. Lukes Medical Center
state: Illinois
city: Bregenz
country: Austria
facility: Novartis Investigative site
city: Vienna
country: Austria
facility: Novartis Investigative site
city: Dresden
country: Germany
facility: Novartis Investigative site
city: Hamburg
country: Germany
facility: Novartis Investigative site
city: Heidelberg
country: Germany
facility: Novartis Investigative site
city: Herne
country: Germany
facility: Novartis Investigative site
city: Kiel
country: Germany
facility: Novartis Investigative site
city: Schweinfurt
country: Germany
facility: Novartis Investigative site
city: Tuebingen
country: Germany
facility: Novartis Investigative site
city: Oslo
country: Norway
facility: Novartis Investigative site
city: Aarau
country: Switzerland
facility: Novartis Investigative site
city: Basel
country: Switzerland
facility: Novartis Investigative site
city: Geneve
country: Switzerland
facility: Novartis Investigative site
city: Lausanne
country: Switzerland
facility: Novartis Investigative site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Walker UA, Tilson HH, Hawkins PN, Poll TV, Noviello S, Levy J, Vritzali E, Hoffman HM, Kuemmerle-Deschner JB; CACZ885D2401 Study Investigators. Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the beta-Confident Registry. RMD Open. 2021 May;7(2):e001663. doi: 10.1136/rmdopen-2021-001663.