Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever

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Brief Title

Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever

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

Brief Summary

      Establish the safety and efficacy of 3 months treatment with canakinumab in patients with
      colchicine resistant Familial Mediterranean Fever.

Study Phase

Phase 2

Study Type


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.

Secondary Outcome

 To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3.


Familial Mediterranean Fever



Study Arms / Comparison Groups



* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

April 2010

Completion Date

August 2011

Primary Completion Date

August 2011

Eligibility Criteria

        Inclusion Criteria:

          -  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)

          -  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

          -  Patients treated with Interferon therapies must complete 1 month washout period.

        Exclusion Criteria:

          -  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




12 Years - 75 Years

Accepts Healthy Volunteers



Novartis Pharmaceuticals, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Novartis Pharmaceuticals

Study Sponsor

Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals

Verification Date

April 2012