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

Novartis

Status and phase

Completed

Phase 2

Conditions

Familial Mediterranean Fever

Treatments

Drug: Canakinumab

Study type

Interventional

Funder types

Industry

Identifiers

CACZ885DTR01

Details and patient eligibility

About

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

Enrollment

10 estimated patients

Sex

All

Ages

12 to 75 years old

Volunteers

No Healthy Volunteers

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