Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients - Full Text View

Purpose

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.

Condition
Cryopyrin-associated Periodic Syndromes (CAPS) Familial Cold Autoinflam Syn (FCAS) Muckle-wells Syn (MWS) Neonatal Onset Multisystem Inflam Disease (NOMID)


Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	An Open-label, Long-term, Prospective, Observational Study to Monitor the Safety and Effectiveness of Ilaris in CAPS Patients

Resource links provided by NLM:

Genetics Home Reference related topics: Muckle-Wells syndrome familial cold autoinflammatory syndrome neonatal onset multisystem inflammatory disease

Drug Information available for: Canakinumab

Genetic and Rare Diseases Information Center resources: Cryopyrin-associated Periodic Syndrome Familial Cold Autoinflammatory Syndrome Wells Syndrome Muckle-Wells Syndrome Chronic Infantile Neurological Cutaneous Articular Syndrome Familial Mediterranean Fever

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

To monitor and further explore the overall safey of canakinumab focusing in serious infections [ Time Frame: At least 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Long-term impact of Ilaris on disease progression (including systemic AA amyloidosis as evidenced by renal function, neurologic and ophthalmologic symptoms, and sensorineural deafness) [ Time Frame: At least 5 years ] [ Designated as safety issue: No ]
Growth and development patterns of children aged 4 to 18 years of age exposed to Ilaris [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]
Identify previously unrecognized serious adverse drug reactions in the treated population [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]
Usage and patterns of dosing of Ilaris in routine clinical practice [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]
Incidence of serious infections [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]
Incidence of malignancies [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]
Incidence of hypersensitivity reactions [ Time Frame: at least 5 years ] [ Designated as safety issue: No ]


Enrollment:	288
Study Start Date:	November 2009
Study Completion Date:	December 2015
Primary Completion Date:	December 2015 (Final data collection date for primary outcome measure)

Eligibility


Ages Eligible for Study:	Child, Adult, Senior
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
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.

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01213641

Locations


United States, Arizona
Little Rock Allergy and Asthma Clinic
Little Rock, Arizona, United States, 72205
United States, Georgia
Allergy Center at Brookstone
Columbus, Georgia, United States, 31904
United States, Illinois
Rush Presbyterian - St. Lukes Medical Center
Chicago, Illinois, United States, 60612
Austria
Novartis Investigative site
Bregenz, Austria
Novartis Investigative site
Vienna, Austria
Germany
Novartis Investigative site
Dresden, Germany
Novartis Investigative site
Hamburg, Germany
Novartis Investigative site
Heidelberg, Germany
Novartis Investigative site
Herne, Germany
Novartis Investigative site
Kiel, Germany
Novartis Investigative site
Schweinfurt, Germany
Novartis Investigative site
Tuebingen, Germany
Norway
Novartis Investigative site
Oslo, Norway
Switzerland
Novartis Investigative site
Aarau, Switzerland
Novartis Investigative site
Basel, Switzerland
Novartis Investigative site
Geneve, Switzerland
Novartis Investigative site
Lausanne, Switzerland

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators


Study Director:	Novartis Pharmaceuticals	Novartis Pharmaceuticals

More Information


Responsible Party:	Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT01213641 History of Changes
Other Study ID Numbers:	CACZ885D2401
Study First Received:	September 30, 2010
Last Updated:	March 25, 2016
Health Authority:	Switzerland: Swissmedic Germany: Paul-Ehrlich-Institut Germany: BfArM

Keywords provided by Novartis:


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)

Additional relevant MeSH terms:


Syndrome Cryopyrin-Associated Periodic Syndromes Disease Pathologic Processes	Hereditary Autoinflammatory Diseases Genetic Diseases, Inborn Skin Diseases, Genetic Skin Diseases

ClinicalTrials.gov processed this record on September 27, 2016

Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients (B-Confident)