Cryopyrin-associated Periodic Syndromes (CAPS)B-ConfidentNCT01213641
Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients
An observational study of Cryopyrin-associated Periodic Syndromes (CAPS) and Familial Cold Autoinflam Syn (FCAS), run by Novartis Pharmaceuticals at 17 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cryopyrin-associated Periodic Syndromes (CAPS), Familial Cold Autoinflam Syn (FCAS), Muckle-wells Syn (MWS)
- How many people
- 288 participants
- Where
- 17 sitesin 5 countries
- Study dates
- Nov 2009to Dec 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Patients receiving Ilaris (canakinumab) at the time of enrollment as part of medical care
Who cannot join
- Local regulations in some locations may exclude patients who receive Ilaris for a non-approved indication
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
17 sites in 5 countries.
17 sites in 5 countries
Germany7 sites
Novartis Investigative site
Dresden
Novartis Investigative site
Hamburg
Novartis Investigative site
Heidelberg
Novartis Investigative site
Herne
Novartis Investigative site
Kiel
Novartis Investigative site
Schweinfurt
Novartis Investigative site
Tübingen
Switzerland4 sites
Novartis Investigative site
Aarau
Novartis Investigative site
Basel
Novartis Investigative site
Geneva
Novartis Investigative site
Lausanne
United States3 sites
Rush Presbyterian - St. Lukes Medical Center
Chicago, Illinois, 60612
Allergy Center at Brookstone
Columbus, Georgia, 31904
Little Rock Allergy and Asthma Clinic
Little Rock, Arizona, 72205
Austria2 sites
Novartis Investigative site
Bregenz
Novartis Investigative site
Vienna
About this study
Official title: An Open-label, Long-term, Prospective, Observational Study to Monitor the Safety and Effectiveness of Ilaris in CAPS Patients
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.
- 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)
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What is being measured
The primary outcome is the main question the study is designed to answer.
To monitor and further explore the overall safey of canakinumab focusing in serious infections
Measured: At least 5 years
Secondary outcomes (7)
Long-term impact of Ilaris on disease progression (including systemic AA amyloidosis as evidenced by renal function, neurologic and ophthalmologic symptoms, and sensorineural deafness)
Measured: At least 5 years
Growth and development patterns of children aged 4 to 18 years of age exposed to Ilaris
Measured: at least 5 years
Identify previously unrecognized serious adverse drug reactions in the treated population
Measured: at least 5 years
Usage and patterns of dosing of Ilaris in routine clinical practice
Measured: at least 5 years
Incidence of serious infections
Measured: at least 5 years
Incidence of malignancies
Measured: at least 5 years
Incidence of hypersensitivity reactions
Measured: at least 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2010 (estimated)
- Study startNov 2009
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cryopyrin-associated Periodic Syndromes (CAPS)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 288 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01213641
- Sponsor's protocol ID
- CACZ885D2401
- Acronym
- B-Confident
- Status last verified
- Mar 2016
- First submitted
- 30 Sep 2010
- Last updated
- 28 Mar 2016
- Results posted
- No
Publications
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.
PubMed 34001647
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Long-term, Prospective, Observational Study to Monitor the Safety and Effectiveness of Ilaris in CAPS Patients
- ClinicalTrials.gov ID
- NCT01213641
- Acronym / sponsor ID
- B-Confident / CACZ885D2401
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 288 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 4 Oct 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- Patients receiving Ilaris (canakinumab) at the time of enrollment as part of medical care
Key exclusion criteria (2 listed)
- Local regulations in some locations may exclude patients who receive Ilaris for a non-approved indication
- Other protocol-defined inclusion/exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To monitor and further explore the overall safey of canakinumab focusing in serious infections
Time frame: At least 5 years
7 secondary outcomes
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
Growth and development patterns of children aged 4 to 18 years of age exposed to Ilaris
Time frame: at least 5 years
Identify previously unrecognized serious adverse drug reactions in the treated population
Time frame: at least 5 years
Usage and patterns of dosing of Ilaris in routine clinical practice
Time frame: at least 5 years
Incidence of serious infections
Time frame: at least 5 years
Incidence of malignancies
Time frame: at least 5 years
Incidence of hypersensitivity reactions
Time frame: at least 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Mar 2016.
- Derived from this study: PubMed 34001647Walker 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.
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NOMID, DIRA
RecruitingPhase 1
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
17 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cryopyrin-associated Periodic Syndromes (CAPS) and Familial Cold Autoinflam Syn (FCAS), run by Novartis Pharmaceuticals at 17 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Germany, Switzerland, United States, Austria, Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To monitor and further explore the overall safey of canakinumab focusing in serious infections (time frame: At least 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01213641, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Mar 2016.