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

Primary Purpose

Familial Mediterranean Fever

Status
Completed
Phase
Phase 2
Locations
Turkey
Study Type
Interventional
Intervention
Canakinumab
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Mediterranean Fever focused on measuring Familial Mediterranean Fever, canakinumab, colchicine resistance

Eligibility Criteria

12 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Istanbul Medical Faculty, Dept of Rheumatology, Capa

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Canakinumab

Arm Description

Outcomes

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.

Secondary Outcome Measures

To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3.
To find the optimal dose of canakinumab for FMF in this population
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
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

Full Information

First Posted
March 16, 2010
Last Updated
April 27, 2012
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01088880
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2012
Overall Recruitment Status
Completed
Study Start Date
April 2010 (undefined)
Primary Completion Date
August 2011 (Actual)
Study Completion Date
August 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Mediterranean Fever
Keywords
Familial Mediterranean Fever, canakinumab, colchicine resistance

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Canakinumab
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Canakinumab
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
To assess the effect of canakinumab with regard to percentage of patients with no attacks in month 3.
Time Frame
12 weeks
Title
To find the optimal dose of canakinumab for FMF in this population
Time Frame
12 weeks
Title
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
Title
To assess PK/PD properties of canakinumab by measuring canakinumab and IL-1beta levels before dosing
Title
To evaluate the safety and tolerability of canakinumab by monitoring adverse events and patient discontinuations due to AE

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Istanbul Medical Faculty, Dept of Rheumatology, Capa
City
Istanbul
Country
Turkey

12. IPD Sharing Statement

Citations:
PubMed Identifier
26337145
Citation
Gul 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(1):243. doi: 10.1186/s13075-015-0765-4.
Results Reference
derived

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

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