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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01088880
Recruitment Status : Completed
First Posted : March 17, 2010
Last Update Posted : May 1, 2012
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

March 16, 2010
March 17, 2010
May 1, 2012
April 2010
August 2011   (Final data collection date for 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 ]
Same as current
Complete list of historical versions of study NCT01088880 on Archive Site
  • 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
Same as current
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Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
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.
Not Provided
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Familial Mediterranean Fever
Drug: Canakinumab
Experimental: Canakinumab
Intervention: Drug: Canakinumab
Gül 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;17:243. doi: 10.1186/s13075-015-0765-4.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
August 2011
August 2011   (Final data collection date for primary outcome measure)

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 contraception
  • 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

Sexes Eligible for Study: All
12 Years to 75 Years   (Child, Adult, Older Adult)
Contact information is only displayed when the study is recruiting subjects
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Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
April 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP