Familial Mediterranean FeverPhase 2NCT01088880
Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
A Phase 2 study testing Canakinumab in people with Familial Mediterranean Fever, run by Novartis Pharmaceuticals at 1 site in Turkey (Türkiye).
Trial summary
Establish the safety and efficacy of 3 months treatment with canakinumab in patients with colchicine resistant Familial Mediterranean Fever. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Familial Mediterranean Fever
- What is being tested?
- Canakinumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- Apr 2010to Aug 2011
- Phase
- Phase 2
- 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 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients between 12 and 75 years of age with active type 1 FMF disease (according to Tel-Hashomer criteria for diagnosis of FMF) despite colchicine therapy (1.5 to 2.0 mg/day).
- Patients who are intolerant to effective doses of colchicine (1.5 to 2 mg/day)
- Patients with demonstrated minimum 1 typical acute attack per month and genetic confirmation of diagnosis (with at least one of the known MEFV gene exon 10 mutations). Patients with manifested amyloidosis are excluded.
- Patients must have a historical data showing a frequency of at least 1 attack/month within the last 3 months before they can be enter the run-in period.
- Patients must have type 1 disease characterized by recurrent and short episodes of inflammation and serositis with an average of at least 1 documented acute FMF attack per month during the previous 6 months and lasting approximately 12 to 72 hours.
- Patients treated with IL-1 therapies must complete washout and have experienced at least 2 attacks since (e.g. Anakinra: 3 day washout; Rilonacept: 3 week washout)
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- Patients treated with anti-TNF drugs must undergo appropriate washout. Prior to randomization, use of Etanercept must be discontinued for 4 weeks or use of Adalimumab or Infliximab must be discontinued for 8 weeks.
- Female subjects of childbearing potential must be using two acceptable methods of contraception
- Patients treated with Interferon therapies must complete 1 month washout period.
Who cannot join
- Patients with end-organ dysfunction due to amyloidosis (e.g. existing biopsy proven amyloidosis or proteinuria > 0.5 gram per day)
- Patients taking steroids within 1 month prior to baseline
- Presence or history of any other inflammatory rheumatic disease
- Positive PPD test (according to local guidance) where a latent or active TB infection cannot be excluded via Quantiferon (T-Spot or radiographic imaging if needed).
- Patients who are pregnant or lactating
- Presence of any active or chronic infection or any major episode of infection requiring hospitalization or treatment with i.v. antibiotics within 30 days or oral antibiotics within 14 days prior to screening
- History or a malignancy within the last 5 years, except for successfully excised squamous or basal cell carcinoma of the skin
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Istanbul Medical Faculty, Dept of Rheumatology, Capa
Istanbul
About this study
Official title: An Open-label, Exploratory Study to Establish the Safety and Efficacy of 3 Months Treatment With Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
Establish the safety and efficacy of 3 months treatment with canakinumab in patients with colchicine resistant Familial Mediterranean Fever.
- Conditions
- Familial Mediterranean Fever
- Being tested
- Canakinumab
- Keywords
- Familial Mediterranean Fever, canakinumab, colchicine resistance
- Medical subject headings
- Familial Mediterranean Fever
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 | Not described Receives: Drug: Canakinumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the effect of canakinumab on the frequency of FMF attacks defined as percentage of patients with at least 50% reduction in the attack frequency during 3 month treatment period.
Measured: 12 weeks
Secondary outcomes (5)
To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3.
Measured: 12 weeks
To find the optimal dose of canakinumab for FMF in this population
Measured: 12 weeks
To assess changes in the severity (acute phase response and VAS evaluation of attack severity by patient) and duration of acute attacks during the treatment period
Measured: 12 weeks
To assess PK/PD properties of canakinumab by measuring canakinumab and IL-1beta levels before dosing
To evaluate the safety and tolerability of canakinumab by monitoring adverse events and patient discontinuations due to AE
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2010 (estimated)
- Study startApr 2010
- Primary completionAug 2011
- Study completionAug 2011
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 Familial Mediterranean FeverStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
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
- NCT01088880
- Sponsor's protocol ID
- CACZ885DTR01
- Status last verified
- Apr 2012
- First submitted
- 16 Mar 2010
- Last updated
- 1 May 2012
- Results posted
- No
Publications
Gul A, Ozdogan H, Erer B, Ugurlu S, Kasapcopur O, Davis N, Sevgi S. Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever. Arthritis Res Ther. 2015 Sep 4;17(1):243. doi: 10.1186/s13075-015-0765-4.
PubMed 26337145
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Exploratory Study to Establish the Safety and Efficacy of 3 Months Treatment With Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
- ClinicalTrials.gov ID
- NCT01088880
- Acronym / sponsor ID
- CACZ885DTR01
- Conditions
- Familial Mediterranean Fever
- Keywords
- Familial Mediterranean Fever, canakinumab, colchicine resistance
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Apr 2010
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 17 Mar 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.
Establish the safety and efficacy of 3 months treatment with canakinumab in patients with colchicine resistant Familial Mediterranean Fever.
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: Canakinumab |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Male and female patients between 12 and 75 years of age with active type 1 FMF disease (according to Tel-Hashomer criteria for diagnosis of FMF) despite colchicine therapy (1.5 to 2.0 mg/day).
- Patients who are intolerant to effective doses of colchicine (1.5 to 2 mg/day)
- Patients with demonstrated minimum 1 typical acute attack per month and genetic confirmation of diagnosis (with at least one of the known MEFV gene exon 10 mutations). Patients with manifested amyloidosis are excluded.
- Patients must have a historical data showing a frequency of at least 1 attack/month within the last 3 months before they can be enter the run-in period.
- Patients must have type 1 disease characterized by recurrent and short episodes of inflammation and serositis with an average of at least 1 documented acute FMF attack per month during the previous 6 months and lasting approximately 12 to 72 hours.
Key exclusion criteria (8 listed)
- Patients with end-organ dysfunction due to amyloidosis (e.g. existing biopsy proven amyloidosis or proteinuria > 0.5 gram per day)
- Patients taking steroids within 1 month prior to baseline
- Presence or history of any other inflammatory rheumatic disease
- Positive PPD test (according to local guidance) where a latent or active TB infection cannot be excluded via Quantiferon (T-Spot or radiographic imaging if needed).
- Patients who are pregnant or lactating
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
To measure the effect of canakinumab on the frequency of FMF attacks defined as percentage of patients with at least 50% reduction in the attack frequency during 3 month treatment period.
Time frame: 12 weeks
5 secondary outcomes
To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3.
Time frame: 12 weeks
To find the optimal dose of canakinumab for FMF in this population
Time frame: 12 weeks
To assess changes in the severity (acute phase response and VAS evaluation of attack severity by patient) and duration of acute attacks during the treatment period
Time frame: 12 weeks
To assess PK/PD properties of canakinumab by measuring canakinumab and IL-1beta levels before dosing
To evaluate the safety and tolerability of canakinumab by monitoring adverse events and patient discontinuations due to AE
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 1 May 2012.
- Derived from this study: PubMed 26337145Gul A, Ozdogan H, Erer B, Ugurlu S, Kasapcopur O, Davis N, Sevgi S. Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever. Arthritis Res Ther. 2015 Sep 4;17(1):243. doi: 10.1186/s13075-015-0765-4.
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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 2 study testing Canakinumab in people with Familial Mediterranean Fever, run by Novartis Pharmaceuticals at 1 site in Turkey (Türkiye).
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 9 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 1 May 2012. 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 Turkey (Türkiye). 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
To measure the effect of canakinumab on the frequency of FMF attacks defined as percentage of patients with at least 50% reduction in the attack frequency during 3 month treatment period. (time frame: 12 weeks). 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 NCT01088880, 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 1 May 2012.