On-demand anakinra for painful attacks in colchicine-resistant FMF patients who refuse daily injections
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
This trial will test whether taking anakinra only when an FMF attack starts reduces pain and symptoms for people with colchicine-resistant FMF who refuse continuous anti‑IL‑1 therapy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Drugs / interventions | canakinumab |
| Locations | 1 site (Paris) |
| Trial ID | NCT06336733 on ClinicalTrials.gov |
What this trial studies
This Phase 3 randomized prospective trial compares on‑demand subcutaneous anakinra (100 mg daily started at prodrome and continued until 24 hours after remission, up to 7 days) plus ongoing colchicine to standard symptomatic analgesic care with ongoing colchicine in colchicine‑resistant FMF patients who decline continuous anti‑IL‑1 therapy. Eligible participants are age 6 and older with confirmed FMF (Livneh criteria and two non‑ambiguous MEFV mutations) and persistent attacks despite maximal tolerated colchicine dose. Patients with active infection, active tuberculosis, recurrent serious infections, or ongoing biological inflammation between attacks are excluded. The study is sponsored by Assistance Publique - Hôpitaux de Paris and conducted at Hopital Tenon in Paris as a randomized interventional comparison of attack‑by‑attack management strategies.
Who should consider this trial
Good fit: Ideal candidates are people aged 6 or older with genetically confirmed FMF and colchicine resistance (≈≥1 attack/month over 3 months on maximum tolerated colchicine) who refuse daily anakinra but have full French ALD coverage and no ongoing inflammation between attacks.
Not a fit: Patients with persistent biological inflammation between attacks, active or recent serious infection, active tuberculosis, recurrent infections, or those who require continuous IL‑1 blockade (for example high amyloidosis risk) are unlikely to benefit from an on‑demand approach.
Why it matters
Potential benefit: If successful, on‑demand anakinra could relieve attack pain and disability for patients who refuse daily injections while reducing overall treatment burden and cost compared with continuous biologic therapy.
How similar studies have performed: Continuous anti‑IL‑1 therapies such as daily anakinra or intermittent canakinumab have shown clinical benefit in colchicine‑resistant FMF, but randomized evidence specifically testing an on‑demand anakinra approach is limited and this is the first randomized prospective comparison.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Paris
- Service de Médecine interne Hopital Tenon — Paris, France (Recruiting)
Study contacts
- Principal investigator: Léa Léa Léa SAVEY — Assistance Publique - Hôpitaux de Paris
- Study coordinator: Léa SAVEY
- Email: lea.savey@aphp.fr
- Phone: 0033 1 56 01 67 91
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.