Cryopyrin Associated Periodic SyndromePhase 3NCT01105507
The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
A Phase 3 study testing canakinumab (company code: ACZ885D) in people with Cryopyrin Associated Periodic Syndrome, run by Novartis Pharmaceuticals at 2 sites in Canada.
Trial summary
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Cryopyrin Associated Periodic Syndrome
- What is being tested?
- canakinumab (company code: ACZ885D)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesCanada
- Study dates
- Aug 2010to 12 May 2012
- Phase
- Phase 3
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients at least 4 years of age at the time of the screening visit
- Patient's informed consent for > or = 18 years of age before any assessment is performed
- Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 18 years of age.
- Patients with a diagnosis of CAPS: FCAS/FCU, MWS, or NOMID/CINCA
- Body weight > or = 15 kg
- Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary)
Who cannot join
- Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at baseline
- Patient of childbearing potential (man or woman) and unwilling to use double barrier method of contraception. It is required that double barrier method of contraception be used (i.e. condom with spermicide or diaphragm with spermicide) by patients of childbearing potential (man or woman) regardless of whether a hormonal agent is also used as a method of contraception.
- Participation in any clinical investigation within 4 weeks prior to dosing
- Live vaccinations within 3 months prior to the start of the trial, during the trial and up to 3 months following the last dose.
- History of significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty).
- History of drug or alcohol abuse within 12 months prior to dosing
- History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
- "Other protocol-defined inclusion/exclusion criteria may apply"
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
Alberta Children's Hospital, Department of Pediatrics
Calgary
Queen Elizabeth II Hospital
Halifax
About this study
Official title: An Open Label Study to Assess the Safety, Tolerability and Efficacy of Canakinumab (ACZ885) in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months.
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Being tested
- canakinumab (company code: ACZ885D)
- Keywords
- Canakinumab, Cryopyrin-associated periodic syndromes, Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria (FCU), Muckle-Wells Syndrome (MWS), Neonatal-Onset Multisystem Inflammatory Disease (NOMID), Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA), Treatment of patients diagnosed with CAPS (Cryopyrin associated periodic syndrome)
- 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.
canakinumab (company code: ACZ885D)Drug
| Study group | Role | What they receive |
|---|---|---|
| canakinumab arm | Experimental | Not described Receives: Drug: canakinumab (company code: ACZ885D) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety & tolerability of canakinumab by measuring the maintenance of response over time using the Investigators Clinical Assessment of Disease Activity scale and the evaluation of serum inflammation markers CRP (C-Reactive Protein), SAA (Serum Amyloid A.
Measured: 18 months
Secondary outcomes (2)
Change in long term maintenance of Health-Related Quality of Life and productivity measured from baseline to end of study.
Measured: Baseline, 18 months
Treatment adherence with canakinumab for the duration of the study.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2010 (estimated)
- Study startAug 2010
- Primary completion12 May 2012
- Study completion12 May 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cryopyrin Associated Periodic SyndromeStudies 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
- 4 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
- NCT01105507
- Sponsor's protocol ID
- CACZ885DCA01
- Status last verified
- Mar 2017
- First submitted
- 25 Mar 2010
- Last updated
- 21 Mar 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Assess the Safety, Tolerability and Efficacy of Canakinumab (ACZ885) in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
- ClinicalTrials.gov ID
- NCT01105507
- Acronym / sponsor ID
- CACZ885DCA01
- Conditions
- Cryopyrin Associated Periodic Syndrome
- Keywords
- Canakinumab, Cryopyrin-associated periodic syndromes, Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria (FCU), Muckle-Wells Syndrome (MWS), Neonatal-Onset Multisystem Inflammatory Disease (NOMID), Chronic Infantile Neurological, Cutaneous
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Aug 2010
- Primary completion
- 12 May 2012 (actual)
- Study completion
- 12 May 2012 (actual)
- First posted
- 16 Apr 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.
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| canakinumab arm | Experimental | Drug: canakinumab (company code: ACZ885D) |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male and female patients at least 4 years of age at the time of the screening visit
- Patient's informed consent for > or = 18 years of age before any assessment is performed
- Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 18 years of age.
- Patients with a diagnosis of CAPS: FCAS/FCU, MWS, or NOMID/CINCA
- Body weight > or = 15 kg
Key exclusion criteria (8 listed)
- Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at baseline
- Patient of childbearing potential (man or woman) and unwilling to use double barrier method of contraception. It is required that double barrier method of contraception be used (i.e. condom with spermicide or diaphragm with spermicide) by patients of childbearing potential (man or woman) regardless of whether a hormonal agent is also used as a method of contraception.
- Participation in any clinical investigation within 4 weeks prior to dosing
- Live vaccinations within 3 months prior to the start of the trial, during the trial and up to 3 months following the last dose.
- History of significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty).
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 (company code: ACZ885D) | Drug | canakinumab arm |
Primary outcome
Safety & tolerability of canakinumab by measuring the maintenance of response over time using the Investigators Clinical Assessment of Disease Activity scale and the evaluation of serum inflammation markers CRP (C-Reactive Protein), SAA (Serum Amyloid A.
Time frame: 18 months
2 secondary outcomes
Change in long term maintenance of Health-Related Quality of Life and productivity measured from baseline to end of study.
Time frame: Baseline, 18 months
Treatment adherence with canakinumab for the duration of the study.
Time frame: 18 months
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 21 Mar 2017.
Related trials for Cryopyrin Associated Periodic Syndrome
RecruitingObservational study
Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases (NOMID/CAPS, DIRA, CANDLE, SAVI, NLRC4-MAS, Still'S-like Diseases, and Other Undifferentiated Autoinflammatory Diseases)
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
Cryopyrin-Associated Periodic Syndromes, Amyloidosis
RecruitingObservational study
Ilaris NIS in Korea
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Recruiting
Circadian Rhythm Deregulation in Patients With CAPS
Cryopyrin Associated Periodic Syndrome, Familial Cold Urticaria
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
2 sites 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 (company code: ACZ885D) in people with Cryopyrin Associated Periodic Syndrome, run by Novartis Pharmaceuticals at 2 sites in Canada.
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 6 inclusion and 8 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 21 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. 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?
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?
Safety & tolerability of canakinumab by measuring the maintenance of response over time using the Investigators Clinical Assessment of Disease Activity scale and the evaluation of serum inflammation markers CRP (C-Reactive Protein), SAA (Serum Amyloid A. (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Mar 2017.