Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean Fever - Full Text View

Purpose

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

Condition	Intervention	Phase
Familial Mediterranean Fever	Drug: Canakinumab	Phase 2

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: familial Mediterranean fever

MedlinePlus related topics: Fever

Drug Information available for: Colchicine Canakinumab

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
To find the optimal dose of canakinumab for FMF in this population [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
To assess PK/PD properties of canakinumab by measuring canakinumab and IL-1beta levels before dosing [ Designated as safety issue: No ]
To evaluate the safety and tolerability of canakinumab by monitoring adverse events and patient discontinuations due to AE [ Designated as safety issue: Yes ]

Estimated Enrollment:	10
Study Start Date:	April 2010
Study Completion Date:	August 2011
Primary Completion Date:	August 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Canakinumab	Drug: Canakinumab

Eligibility

Ages Eligible for Study:	12 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01088880

Locations

Turkey
Istanbul Medical Faculty, Dept of Rheumatology, Capa
Istanbul, Turkey

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

No publications provided

Responsible Party:	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT01088880 History of Changes
Other Study ID Numbers:	CACZ885DTR01
Study First Received:	March 16, 2010
Last Updated:	April 27, 2012
Health Authority:	Turkey: Ministry of Health Turkey: Ethics Committee

Keywords provided by Novartis:

Familial Mediterranean Fever
canakinumab
colchicine resistance

Additional relevant MeSH terms:

Brucellosis
Fever
Familial Mediterranean Fever
Hereditary Autoinflammatory Diseases
Gram-Negative Bacterial Infections
Bacterial Infections
Body Temperature Changes
Signs and Symptoms
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Colchicine

Antibodies, Monoclonal
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Gout Suppressants
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 22, 2013