Randomized Controlled Trial in Patients on Long-term Colchicine With Colchicine-resistant Familial Mediterranean Fever (FMF) to Evaluate the Efficacy of On-demand Anakinra Treatment for Painful Attacks in Patients Who Refuse Continuous Daily Therapy

APHP220828

To evaluate the efficacy on clinical symptoms in case of FMF attack among FMF patients resistant to Colchicine of * on demand anakinra treatment (100 mg/d from the prodromal phase of the attack until 24 hours of remission (during 7 days maximum) associated with daily colchicine. * compared to analgesic associated with daily colchicine in patients refusing continuous anti-IL-1 treatment.

2024-03-01

2026-03-03

2026-09-15

Not yet recruiting

Early phase I

50

Inclusion Criteria: * Age \>= 6 years old with no upper limit * Proven FMF according to Livneh international criteria and 2 non ambiguous MEFV mutations. * Colchicine resistance defined as persistent FMF attack despite the maximum daily posology of colchicine (average one or more attacks per month over a 3-months period) FMF Attack is defined by: * Arthritis or * Chest pain or * Abdominal pain or * Myalgia or * Erysipelas-like skin lesion Duration of episodes 1-4 days. * Patient refusing daily anakinra injections- * Patients covered at 100% by the health insurance (ALD) * Patient who do not have biological inflammation between attacks * Written informed consent of the patients and or his legal representatives Exclusion Criteria: * Evidence of active tuberculosis * Infection requiring treatment with intravenous antibiotics within 2 weeks prior to inclusion * 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) * Contraindication to anakinra (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 * Patients with neutropenia (ANC \<1.5 x 10\^9/l) * Inability to provide informed consent * Ongoing chronic treatment with anti IL1 biotherapy since at least 3 months * Pregnant women * Women in labor and nursing mothers * Patients in emergency situations and people hospitalized without consent * No health care insurance * Contraindication to colchicine * Patient participating in another interventional clinical trial * Patient deprived of liberty * Patient under guardianship or curatorship * Patient under court protection Randomization criteria : * Absence of active or latent tuberculosis (no tuberculosis sign on chest X-ray and a negative quantiferon) * Negative pregnancy test

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}

2024-03-29