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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01088880
• Date of registration: 16/03/2010
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever
• Scientific 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
• Date of first enrolment: April 2010
• Target sample size: 10
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01088880
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Turkey

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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
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



Age minimum: 12 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Familial Mediterranean Fever

Intervention(s)

Drug: Canakinumab

Primary Outcome(s)

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]

Secondary Outcome(s)

To assess PK/PD properties of canakinumab by measuring canakinumab and IL-1beta levels before dosing [Time Frame: No]

To evaluate the safety and tolerability of canakinumab by monitoring adverse events and patient discontinuations due to AE [Time Frame: Yes]

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 the effect of canakinumab with regard to percentage of patients with no attacks in month 3. [Time Frame: 12 weeks]

Secondary ID(s)

CACZ885DTR01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors