Anakinra for Familial Mediterranean Fever

Inclusion criteria

• {"criterion_text":"- Age > 6 years old with no upper limit."}
• {"criterion_text":"- Proven FMF according to Livneh international criteria (2) and 2 non ambiguous MEFV mutations."}
• {"criterion_text":"- Colchicine resistance defined as persistent FMF attack despite the maximum daily posology of colchicine (average one or more attacks per month over a 3-month period)."}
• {"criterion_text":"- FMF Attack is defined by: -\tArthritis"}
• {"criterion_text":"- FMF Attack is defined by: -\tChest pain"}
• {"criterion_text":"- FMF Attack is defined by: -\tAbdominal pain"}
• {"criterion_text":"- FMF Attack is defined by: -\tMyalgia"}
• {"criterion_text":"- FMF Attack is defined by: -\tErysipelas-like skin lesion"}
• {"criterion_text":"- Duration of episodes 1–4 days."}
• {"criterion_text":"- Patient refusing daily anakinra injections"}
• {"criterion_text":"- Patients covered at 100% by the health insurance (ALD)"}
• {"criterion_text":"- Patients who do not have biological inflammation between attacks"}
• {"criterion_text":"- Written informed consent of the patients and or his legal representatives"}

Exclusion criteria

• {"criterion_text":"- Evidence of active tuberculosis"}
• {"criterion_text":"- Infection requiring treatment with intravenous antibiotics within 2 weeks prior to Inclusion"}
• {"criterion_text":"- History of recurrent infection (Need more than 4 courses of antibiotic treatment per year (in children) or more >2 times per year (in adults), experience pneumonia twice over any time or > 3 bacterial sinusitis in 1 year)"}
• {"criterion_text":"- Contraindication to anakinra :"}
• {"criterion_text":"-Hypersensitivity to the active substance or to any of the excipients (Citric acid, anhydrous Sodium chloride, Disodium edetate dehydrate, Polysorbate 80, Sodium hydroxide, Water for injections ) or to E. coli derived proteins"}
• {"criterion_text":"- Patients with neutropenia (ANC <1.5 x 109/l)"}
• {"criterion_text":"- Contraindication to colchicine"}
• {"criterion_text":"- Inability to provide informed consent"}
• {"criterion_text":"- Ongoing chronic treatment with anti IL1 biotherapy since at least 3 months"}
• {"criterion_text":"- Pregnant women or breast feeding"}
• {"criterion_text":"- No health care insurance"}
• {"criterion_text":"- Patient participating in another interventional clinical trial"}
• {"criterion_text":"- Patient deprived of liberty"}
• {"criterion_text":"- Patient under guardianship or curatorship"}

Primary endpoints

• {"endpoint_text":"- Mean number of painful days per month at 6 months of treatment.","definition_or_measurement_approach":"Mean number of painful days per month at 6 months of treatment."}

Secondary endpoints

• {"endpoint_text":"- Efficacy: −Cumulative days of FMF attack treatment from randomization to 6 months, - AIDAI (Auto-inflammatory Diseases activity index) score D0 and M6 Number of painful days and severity of FMF attacks (measured by VAS Visual Analogue Scale) occurring between randomization and M6 − Quality of life score measured by EuroQOL questionnaire (EQ-5D5L) at M6","definition_or_measurement_approach":"Cumulative days of FMF attack treatment from randomization to 6 months; AIDAI score at D0 and M6; number of painful days and severity measured by VAS between randomization and M6; quality of life measured by EQ-5D-5L at specified visits (J0, M1, M3, M6)."}
• {"endpoint_text":"- Safety: − Number of local cutaneous reaction at 6 months (erythema and oedema involving the injection sites) in the anakinra arm − Proportion of adverse events","definition_or_measurement_approach":"Number of local cutaneous reactions at 6 months (erythema and oedema at injection sites) in the anakinra arm; proportion of adverse events."}

France

Earliest CTIS Part Ii Submission Date

18-12-2023

Latest Decision Or Authorization Date

30-10-2024

Processing Time Days

317

Number Of Sites

11

Number Of Participants

50

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France