Familial Mediterranean Fever
Conditions
Interventions
Anakinra:Anakinra
Sponsors
Beijing Childrens Hospital,Capital Medical University
Eligibility
Sex/Gender
All
Age
2 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Informed consent form signed by the patient or a legal guardian representative. 2. Male or female patients, 2 years of age or older with a body weight = 10 kg. 3. Diagnosis of FMF (adapted Tel Hashomer criteria) [62], confirmed by a positive genetic testing i.e., mutations in both alleles of the MEFV gene (i.e. homozygous or compound heterozygous) 4. Patient must have an estimated mean of at least 2 acute FMF attacks per month within 2 months prior to enrollment to the study. 5. Patient must be resistant to colchicine treatment. a. Definition of colchicine-resistant FMF: Colchicine resistance is defined by = 1 attack per month in any of the 4 FMF sites (abdomen, chest, joints, or skin), in compliant patients receiving the maximally tolerated dose of colchicine (= 2 to = 3 mg/day) for = 6 months [4]. 6. Female patients of childbearing potential and male patients with female partners of childbearing potential must use an effective method of contraception during the study (abstinence being a possible option) as well as a negative pregnancy test prior to enrollment for females of childbearing potential and participating in the study. 7. Negative tuberculosis screening confirmed at screening visit by the Mantoux Tuberculin skin test (TST) using purified protein derivative (PPD), or by Interferon-Gamma-Release Assays (IGRAs) e.g., QuantiFERON® TB Gold Plus (QFT-Plus) or T-Spot® (TB Test) within 8 weeks prior to enrollment. Negative results must be complemented by the medical history, physical examination, and Chest X-Ray. Patients presenting positive TST or IGRA with or without active or clinical suspicion of latent tuberculosis are not eligible to enter the study. Previously vaccinated for Tuberculosis patients: IGRA positive patients are not eligible to enter the Study; 1.TST positive patients with an induration of 15 mm and more are also not eligible to enter the study, TST positive patients (with an induration up to 15 mm) are also not eligible to enter the study, unless an IGRA test is subsequently performed and provides a negative result.
Exclusion criteria
Exclusion criteria: 1. Previous enrollment to this study. 2. Participation in another clinical interventional study 30 days prior to enrollment. 3. Treatment with an investigational drug within 5 half-lives prior to enrollment. 4. Previous or current treatment with anakinra, or any other IL-1 inhibitor. However, previous treatment with canakinumab or rilonacept is allowed if canakinumab, or rilonacept, was discontinued for reasons other than lack of efficacy and after a washout period of minimum 130 days for canakinumab and 35 days in the case for rilonacept. Patients who have discontinued canakinumab, or rilonacept, because of insufficient effect or refractory disease are not allowed to be enrolled in the study. 5. Use of the following therapies prior to enrollment: • Narcotic analgesics within 24 hours prior to enrollment. • Dapsone or etanercept within 2 weeks prior to enrollment. • Intraarticular, intramuscular, or intravenous administration of glucocorticoids within 72h (3 days) prior to enrollment, or intravenous immunoglobulin within 4 weeks prior to enrollment. • Leflunomide within 10 weeks prior to enrollment; 1). infliximab within 8 weeks prior to enrollment; 2). adalimumab within 15 weeks prior to enrollment. • Thalidomide within 72h (3 days) prior to enrollment; 3). cyclosporine, within 5 days prior to enrollment; 4). mycophenolate mofetil within 1 week prior to enrollment; 5). 6-mercaptopurine within 25 days prior to enrollment; 6). azathioprine within 72h (3 days) prior to enrollment; 7). cyclophosphamide within 96h (4 days) prior to enrollment; 8).chlorambucil within 48h (2 days) prior to enrollment; 9).tofacitinib within 24 hours (1 day), baricitinib within 72 h (3 days) and any other JAK inhibitor within 5 half-lives prior to enrollment; 10).methotrexate within 1 week (7 days) prior to enrollment and any other immunosuppressant within 12 weeks prior to enrollment. • Tocilizumab within 16 weeks prior to enrollment or any other immunomodulatory medication within 5 half-lives prior to enrollment. Rituximab within 13 weeks prior to enrollment. 6. Vaccination with a live vaccine within 4 weeks prior to enrollment. 7. Known presence or suspicion of active, chronic, or recurrent bacterial, fungal, or viral infections, including but not limited to tuberculosis, HIV infection, Covid-19 infection, hepatitis B or C infection at baseline. 8. Clinical evidence of liver disease or liver injury as indicated by presence of abnormal liver tests: a. AST or ALT > 5 x ULN, or b. AST or ALT > 3 x ULN and elevated bilirubin > 2 x ULN. 9. Presence of severe chronic kidney disease (CKD) stages 4 and 5 (estimated creatinine clearance < 30 mL/min/1.73m2). 10. Diagnosis of amyloidosis at baseline. 11. Presence of neutropenia (ANC < 1.5 x 109/L). 12. Presence of thrombocytopenia (platelets count < 100 x 109/L). 13. History of malignancy within 5 years prior to enrollment. Exceptions are basal cell skin cancer, carcinoma-in-situ of the cervix, or low-risk prostate cancer after curative therapy. 14. History of autoimmune and other autoinflammatory diseases: e.g. diabetes mellitus type 1, gastrointestinal diseases such as Crohn’s disease, ulcerative colitis, and irritable bowel syndrome, active ischemic cardiovascular disease, congestive heart failure, and chronic obstructive pulmonary disease. 15. Known hypersensitivity to E. Coli-derived proteins, or any components of anakinra. 16. Pregnant or lactating women. 17. Foreseeable inability to cooperate with
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The change in the number of FMF attacks per month per patient from baseline to subsequent study visits, up to Month 6. The frequency of FMF attacks at baseline is based on the mean number of FMF attacks per month occurring within 2 months prior to study drug initiation, as reported by the patient. ; | — |
Countries
China
Contacts
Public ContactLi Caifeng
Beijing Childrens Hospital,Capital Medical University
Outcome results
None listed