NOMIDPhase 3NCT00770601
Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease
A Phase 3 study testing Canakinumab in people with NOMID and CINCA Syndrome, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous, articular (CINCA) syndrome. Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- NOMID, CINCA Syndrome
- What is being tested?
- Canakinumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Jan 2009to 17 Feb 2011
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
None listed.
Who cannot join
- Subjects meeting any of the following criteria will be excluded from entry into or continuation in the study unless sponsor approval is obtained:
- Pregnant or breastfeeding women.
- Participation in any clinical trial investigation within 4 weeks prior to dosing or longer if required by local regulation, with the exception of trials with anakinra and/or canakinumab.
- In case of previous treatment with biologic agents or DMARDs, an appropriate washout period (as according to the recognized duration of effect and half lives) will be required for such patients to be eligible to participate in the trial, e.g.:
- Previous treatment and required washout period prior to baseline and thereafter:
- Rituximab, 26 weeks
Show all 27Show fewer
- Infliximab, 12 weeks
- Adalimumab, 8 weeks
- Etanercept, 4 weeks
- Anakinra, 1 day
- Any other investigational biologics, 8 weeks
- Leflunomide, 4 weeks
- Thalidomide, 4 weeks
- Cyclosporine, 4 weeks
- i.v. immunoglobulin (i.v. Ig), 8 weeks
- Dapsone, mycophenolate mofetil, 3 weeks
- Corticosteroids greater than or equal to 20 mg/day or greater than 0.4 mg/kg prednisone or prednisone equivalent, whichever applies, 1 week
- Donation or loss of 300 mL or more of blood within 8 weeks prior to dosing.
- A past medical history of clinically significant ECG abnormalities or a family history of a prolonged QT-interval syndrome.
- History of immunocompromise.
- Positive test for or prior history of HIV (ELISA and Western blot), Hepatitis B (Hepatitis B surface antigen) or Hepatitis C.
- Presence of active infections or a history of pulmonary TB infection with or without documented adequate therapy. Subjects with current active TB, or recent close exposure to an individual with active TB are excluded from the study.
- Presence of any additional rheumatic disease or significant systemic disease. For example, major chronic infectious/ inflammatory/ immunologic disease (such as inflammatory bowel disease, psoriatic arthritis, spondyloarthropathy, systemic lupus erythematosus in addition to the autoinflammatory disease).
- Treatment with a live virus vaccine during 3 months prior to baseline visit. No live vaccines will be allowed throughout the course of this study and up to 3 months following the last dose.
- History of renal transplant.
- History of malignancy. Subjects deemed cured of superficial malignancies such as cutaneous basal or squamous cell carcinomas, or in situ cervical cancer may be enrolled.
- Presence of any of the following laboratory abnormalities: ALT or AST greater than 2 times the upper limit of normal (ULN), platelet count less than 100x10(9)/L.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), 9
Bethesda, Maryland, 20892
About this study
Official title: A Multi-Center, Open Label, 24-Month Treatment Study to Establish the Safety, Tolerability, Efficacy, Pharmacokinetics of Canakinumab (Anti-IL-1 Beta Antibody) in Patients With NOMID / CINCA Syndrome
This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous, articular (CINCA) syndrome. This disease can cause rash, joint deformities, brain inflammation, problems with the eyes and learning difficulties. Canakinumab is an experimental drug that inhibits the action of a protein produced by the body called human IL-1beta, which is responsible for the symptoms in NOMID and also contributes to many other kinds of inflammatory diseases.
Patients 2 years of age and older with NOMID / CINCA may be eligible for this study. Participants undergo the following procedures:
Read more
Screening Phase
- Medical history and review of medical records
- Blood tests
- Daily diary of symptoms and medicines take
Washout/Lead-in Phase
- Discontinuation of anakinra or other medications, a 6 to 48-hour run-in period (only for patients who discontinued anakinra or other IL-1 blocking therapy).
Treatment Phase
- Injection of canakinumab under the skin every 8 weeks for 6 months
- Monitoring and evaluations during treatment, including:
- Quality-of-life questionnaires and daily diary
- Vital signs measurements (heartbeat, blood pressure, temperature)
- Blood tests
- Electrocardiogram
- Tuberculosis skin test
- Neurological, eye and skin examinations at beginning and end of study
- Cognitive evaluation at beginning and end of study
- Lumbar puncture (spinal tap) at the beginning of the study, 2 weeks after the second dosing of canakinumab and at the end of the study
- X-rays and bone density scan at beginning and end of study
- Magnetic resonance imaging (MRI) of the head at beginning and end of study
Follow-up Phase
- Monthly clinic visits after the last dose of canakinumab for a minimum of 60 days
End-of-Study Evaluation
- Series of tests 8 weeks after last dose of canakinumab to evaluate treatment response and side effects
Read the detailed description
This open-label study was designed to investigate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of canakinumab, a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody, in patients with NOMID / CINCA syndrome. A total of 25 to 30 patients will be enrolled into the study. The study consists of two stages:
In the first pilot stage it is planned to enroll 5 patients undergoing PK/PD assessments in blood and cerebrospinal fluid (CSF), and monitoring of efficacy (CNS relapse and inflammatory relapse) to confirm the dose and dosing regimen before enlarging the number of patients enrolled into the study. Up to 5 additional patients may be enrolled in this stage if the variability of the responses to treatment is high. Interim analyses will be conducted as required. Following Stage 1, a second confirmatory stage will be conducted, enrolling 20 additional patients. In this stage patients will be treated with the dose and dosing regimen based on the assessment of the efficacy profile in Stage 1 and exploratory PK/PD assessment. In each stage there will be a 3-week screening period to collect pre-treatment parameters, a run-in period (only for patients who discontinue anakinra), a baseline evaluation prior to each drug administration, a 24-week treatment period with fixed dosing of canakinumab, and a study completion visit. Patients whose body weight is greater than 40 kg will receive canakinumab 150 mg as a subcutaneous (s.c.) injection, and patients with a body weight less than or equal to 40 kg will receive canakinumab 2 mg/kg s.c. Patients will be administered canakinumab every 8 weeks and will undergo an observation period after each dose administration in order to evaluate the response to treatment. Patients who do not achieve complete remission following canakinumab injection in any treatment period will be re-dosed within Day 15 or receive the following dose adjustments:
- 300 mg s.c. (or 4 mg/kg for patients with a body weight less than or equal to 40 kg)
- 450 mg s.c. (or 6 mg/kg for patients with a body weight less than or equal to 40 kg)
- 600 mg s.c. (or 8 mg/kg for patients with a body weight less than or equal to 40 kg)
End of Study will occur when patients discontinue from the study or complete this study. The End of Study visit should occur 8 weeks (plus or minus 1 week) after the last injection. All patients who complete the 6-month evaluation may be offered to enter an extension study (CACZ885D2201E1) conducted to assess long term safety and efficacy data.
- Conditions
- NOMID, CINCA Syndrome
- Being tested
- Canakinumab
- Keywords
- NOMID, CINCA Syndrome, Canakinumab
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CanakinumabDrug
| Study group | Role | What they receive |
|---|---|---|
| Canakinumab | Experimental | Participants received body-weight stratified dosage of canakinumab treatment at 300 milligrams (mg) (for participants weighing more than 40 kilograms (kg)) and at 2 mg/kg (for participants weight less than or equal to 40 kg) subcutaneously every 4-8 weeks as per investigator discretion for a treatment period of 6 months. The first 3 Neonatal-Onset Multisystem Inflammatory Disease (NOMID) participants enrolled received a dose of 150 mg (>40 kg) and 2 mg/kg for children <40 kg. Since this dose was insufficient to fully control the symptoms of the disease the 300 mg / 4mg/kg dose was introduced by Protocol Amendment 2. Receives: Drug: Canakinumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Complete Remission and Relapse After 6 Months of Canakinumab Treatment.
The primary endpoint of the study was the percentage of participants experiencing a relapse Central nervous system (CNS) relapse and/or inflammatory relapse) during 6-month open label administration of canakinumab in participant with NOMID/Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA). Complete remission consisted of inflammatory remission and CNS remission. 1 Inflammatory (systemic) remission was defined as follows (all criteria to be fulfilled): -Serum C-reactive protein (CRP) AND serum amyloid A (SAA) ≤ 10 milligrams/litre AND -daily diary score (mean score/week) ≤ 2. 2) CNS remission was defined as follows: Headache score (from the daily diary, mean score/week) < 0.5 AND, when a lumbar puncture was performed: Normal values of white cell count (WBC) (≤15 cells/mm3) in CSF.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2008 (estimated)
- Study start26 Jan 2009
- Primary completion17 Feb 2011
- Study completion17 Feb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for NOMIDStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00770601
- Sponsor's protocol ID
- CACZ885D2201
- Other IDs
- 09-AR-0006
- Status last verified
- Jun 2021
- First submitted
- 9 Oct 2008
- Last updated
- 10 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Goldbach-Mansky R, Dailey NJ, Canna SW, Gelabert A, Jones J, Rubin BI, Kim HJ, Brewer C, Zalewski C, Wiggs E, Hill S, Turner ML, Karp BI, Aksentijevich I, Pucino F, Penzak SR, Haverkamp MH, Stein L, Adams BS, Moore TL, Fuhlbrigge RC, Shaham B, Jarvis JN, O'Neil K, Vehe RK, Beitz LO, Gardner G, Hannan WP, Warren RW, Horn W, Cole JL, Paul SM, Hawkins PN, Pham TH, Snyder C, Wesley RA, Hoffmann SC, Holland SM, Butman JA, Kastner DL. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition. N Engl J Med. 2006 Aug 10;355(6):581-92. doi: 10.1056/NEJMoa055137.
PubMed 16899778Hawkins PN, Lachmann HJ, Aganna E, McDermott MF. Spectrum of clinical features in Muckle-Wells syndrome and response to anakinra. Arthritis Rheum. 2004 Feb;50(2):607-12. doi: 10.1002/art.20033.
PubMed 14872505Hirsch E, Irikura VM, Paul SM, Hirsh D. Functions of interleukin 1 receptor antagonist in gene knockout and overproducing mice. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):11008-13. doi: 10.1073/pnas.93.20.11008.
PubMed 8855299Sibley CH, Chioato A, Felix S, Colin L, Chakraborty A, Plass N, Rodriguez-Smith J, Brewer C, King K, Zalewski C, Kim HJ, Bishop R, Abrams K, Stone D, Chapelle D, Kost B, Snyder C, Butman JA, Wesley R, Goldbach-Mansky R. A 24-month open-label study of canakinumab in neonatal-onset multisystem inflammatory disease. Ann Rheum Dis. 2015 Sep;74(9):1714-9. doi: 10.1136/annrheumdis-2013-204877. Epub 2014 Jun 6.
PubMed 24906637
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Open Label, 24-Month Treatment Study to Establish the Safety, Tolerability, Efficacy, Pharmacokinetics of Canakinumab (Anti-IL-1 Beta Antibody) in Patients With NOMID / CINCA Syndrome
- ClinicalTrials.gov ID
- NCT00770601
- Acronym / sponsor ID
- CACZ885D2201
- Conditions
- NOMID, CINCA Syndrome
- Keywords
- NOMID, CINCA Syndrome, Canakinumab
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- Study start
- 26 Jan 2009 (actual)
- Primary completion
- 17 Feb 2011 (actual)
- Study completion
- 17 Feb 2011 (actual)
- First posted
- 10 Oct 2008 (estimated)
- Results
- Posted 10 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous, articular (CINCA) syndrome. This disease can cause rash, joint deformities, brain inflammation, problems with the eyes and learning difficulties. Canakinumab is an experimental drug that inhibits the action of a protein produced by the body called human IL-1beta, which is responsible for the symptoms in NOMID and also contributes to many other kinds of inflammatory diseases.
Patients 2 years of age and older with NOMID / CINCA may be eligible for this study. Participants undergo the following procedures:
Screening Phase
- Medical history and review of medical records
- Blood tests
- Daily diary of symptoms and medicines take
Washout/Lead-in Phase
- Discontinuation of anakinra or other medications, a 6 to 48-hour run-in period (only for patients who discontinued anakinra or other IL-1 blocking therapy).
Treatment Phase
- Injection of canakinumab under the skin every 8 weeks for 6 months
- Monitoring and evaluations during treatment, including:
- Quality-of-life questionnaires and daily diary
- Vital signs measurements (heartbeat, blood pressure, temperature)
- Blood tests
- Electrocardiogram
- Tuberculosis skin test
- Neurological, eye and skin examinations at beginning and end of study
- Cognitive evaluation at beginning and end of study
- Lumbar puncture (spinal tap) at the beginning of the study, 2 weeks after the second dosing of canakinumab and at the end of the study
- X-rays and bone density scan at beginning and end of study
- Magnetic resonance imaging (MRI) of the head at beginning and end of study
Follow-up Phase
- Monthly clinic visits after the last dose of canakinumab for a minimum of 60 days
End-of-Study Evaluation
- Series of tests 8 weeks after last dose of canakinumab to evaluate treatment response and side effects
Detailed description
This open-label study was designed to investigate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of canakinumab, a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody, in patients with NOMID / CINCA syndrome. A total of 25 to 30 patients will be enrolled into the study. The study consists of two stages:
In the first pilot stage it is planned to enroll 5 patients undergoing PK/PD assessments in blood and cerebrospinal fluid (CSF), and monitoring of efficacy (CNS relapse and inflammatory relapse) to confirm the dose and dosing regimen before enlarging the number of patients enrolled into the study. Up to 5 additional patients may be enrolled in this stage if the variability of the responses to treatment is high. Interim analyses will be conducted as required. Following Stage 1, a second confirmatory stage will be conducted, enrolling 20 additional patients. In this stage patients will be treated with the dose and dosing regimen based on the assessment of the efficacy profile in Stage 1 and exploratory PK/PD assessment. In each stage there will be a 3-week screening period to collect pre-treatment parameters, a run-in period (only for patients who discontinue anakinra), a baseline evaluation prior to each drug administration, a 24-week treatment period with fixed dosing of canakinumab, and a study completion visit. Patients whose body weight is greater than 40 kg will receive canakinumab 150 mg as a subcutaneous (s.c.) injection, and patients with a body weight less than or equal to 40 kg will receive canakinumab 2 mg/kg s.c. Patients will be administered canakinumab every 8 weeks and will undergo an observation period after each dose administration in order to evaluate the response to treatment. Patients who do not achieve complete remission following canakinumab injection in any treatment period will be re-dosed within Day 15 or receive the following dose adjustments:
- 300 mg s.c. (or 4 mg/kg for patients with a body weight less than or equal to 40 kg)
- 450 mg s.c. (or 6 mg/kg for patients with a body weight less than or equal to 40 kg)
- 600 mg s.c. (or 8 mg/kg for patients with a body weight less than or equal to 40 kg)
End of Study will occur when patients discontinue from the study or complete this study. The End of Study visit should occur 8 weeks (plus or minus 1 week) after the last injection. All patients who complete the 6-month evaluation may be offered to enter an extension study (CACZ885D2201E1) conducted to assess long term safety and efficacy data.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Canakinumab | Experimental | Drug: CanakinumabParticipants received body-weight stratified dosage of canakinumab treatment at 300 milligrams (mg) (for participants weighing more than 40 kilograms (kg)) and at 2 mg/kg (for participants weight less than or equal to 40 kg) subcutaneously every 4-8 weeks as per investigator discretion for a treatment period of 6 months. The first 3 Neonatal-Onset Multisystem Inflammatory Disease (NOMID) participants enrolled received a dose of 150 mg (>40 kg) and 2 mg/kg for children <40 kg. Since this dose was insufficient to fully control the symptoms of the disease the 300 mg / 4mg/kg dose was introduced by Protocol Amendment 2. |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (27 listed)
- Subjects meeting any of the following criteria will be excluded from entry into or continuation in the study unless sponsor approval is obtained:
- Pregnant or breastfeeding women.
- Participation in any clinical trial investigation within 4 weeks prior to dosing or longer if required by local regulation, with the exception of trials with anakinra and/or canakinumab.
- In case of previous treatment with biologic agents or DMARDs, an appropriate washout period (as according to the recognized duration of effect and half lives) will be required for such patients to be eligible to participate in the trial, e.g.:
- Donation or loss of 300 mL or more of blood within 8 weeks prior to dosing.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Canakinumab | Drug | Canakinumab |
Primary outcome
Percentage of Participants With Complete Remission and Relapse After 6 Months of Canakinumab Treatment.
Time frame: 6 months
The primary endpoint of the study was the percentage of participants experiencing a relapse Central nervous system (CNS) relapse and/or inflammatory relapse) during 6-month open label administration of canakinumab in participant with NOMID/Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA). Complete remission consisted of inflammatory remission and CNS remission. 1 Inflammatory (systemic) remission was defined as follows (all criteria to be fulfilled): -Serum C-reactive protein (CRP) AND serum amyloid A (SAA) ≤ 10 milligrams/litre AND -daily diary score (mean score/week) ≤ 2. 2) CNS remission was defined as follows: Headache score (from the daily diary, mean score/week) < 0.5 AND, when a lumbar puncture was performed: Normal values of white cell count (WBC) (≤15 cells/mm3) in CSF.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 16899778Goldbach-Mansky R, Dailey NJ, Canna SW, Gelabert A, Jones J, Rubin BI, Kim HJ, Brewer C, Zalewski C, Wiggs E, Hill S, Turner ML, Karp BI, Aksentijevich I, Pucino F, Penzak SR, Haverkamp MH, Stein L, Adams BS, Moore TL, Fuhlbrigge RC, Shaham B, Jarvis JN, O'Neil K, Vehe RK, Beitz LO, Gardner G, Hannan WP, Warren RW, Horn W, Cole JL, Paul SM, Hawkins PN, Pham TH, Snyder C, Wesley RA, Hoffmann SC, Holland SM, Butman JA, Kastner DL. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition. N Engl J Med. 2006 Aug 10;355(6):581-92. doi: 10.1056/NEJMoa055137.
- Background: PubMed 14872505Hawkins PN, Lachmann HJ, Aganna E, McDermott MF. Spectrum of clinical features in Muckle-Wells syndrome and response to anakinra. Arthritis Rheum. 2004 Feb;50(2):607-12. doi: 10.1002/art.20033.
- Background: PubMed 8855299Hirsch E, Irikura VM, Paul SM, Hirsh D. Functions of interleukin 1 receptor antagonist in gene knockout and overproducing mice. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):11008-13. doi: 10.1073/pnas.93.20.11008.
- Derived from this study: PubMed 24906637Sibley CH, Chioato A, Felix S, Colin L, Chakraborty A, Plass N, Rodriguez-Smith J, Brewer C, King K, Zalewski C, Kim HJ, Bishop R, Abrams K, Stone D, Chapelle D, Kost B, Snyder C, Butman JA, Wesley R, Goldbach-Mansky R. A 24-month open-label study of canakinumab in neonatal-onset multisystem inflammatory disease. Ann Rheum Dis. 2015 Sep;74(9):1714-9. doi: 10.1136/annrheumdis-2013-204877. Epub 2014 Jun 6.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- link to the results on Novartis results databaseLink provided by the sponsor.
Related trials for NOMID
RecruitingObservational study
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Canakinumab in people with NOMID and CINCA Syndrome, run by Novartis Pharmaceuticals at 1 site in United States.
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 0 inclusion and 27 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: stopped early, as of the sponsor's last update on 10 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Complete Remission and Relapse After 6 Months of Canakinumab Treatment. (time frame: 6 months). 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 NCT00770601, which also lists 4 linked publications.
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 10 Jun 2021.