AOSD, FMF, CAPS, and Schnitzler syndrome
Conditions
Interventions
Cryopyrin-Associated Periodic Syndrome (CAPS):Firsekibart
Adult-onset Still's disease (AOSD):Firsekibart
Familial Mediterranean Fever (FMF):Firsekibart
Schnitzler Syndrome:Firsekibart
Sponsors
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Written informed consent form signed by the subject must have been obtained. 2. Willing and committed to return to the study site to complete all study visits and all study-related procedures. 3. Male and female subjects aged >= 18 years and = 2, considered to be in active disease. (2): CAPS: Subjects meeting the diagnostic criteria for CAPS, with NLRP3 mutation and requiring treatment, and a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. (3): Schnitzler syndrome: Subjects meeting the diagnostic criteria for SchS, with a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. (4): FMF: Subjects meeting the Tel Hashomer diagnostic criteria (1997), with a prior attack frequency of at least 1 per month. For patients receiving biologic therapy, this criterion applies to the last 12 months before initiation of any biologic therapy, and a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. And meeting one of the following: Active disease despite colchicine treatment (at least 1.5 mg to 3.0 mg/day). Patients currently on colchicine will remain on a stable dose during the study. Colchicine intolerance (at least 1.5 mg to 3.0 mg/day). 5. .If receiving NSAID therapy: only one NSAID at a stable dose for >= 1 week before enrollment (dose <= maximum recommended daily dose) is allowed (excluding temporary antipyretic use with <= 1 dose per day and <= 2 cumulative days within 2 weeks before dosing), and the dose will not be increased during the study. 6. .If receiving glucocorticoid therapy: stable dose <= 10 mg/day (prednisolone or equivalent) for at least 2 weeks before enrollment, and the dose will not be increased during the study. 7. If receiving disease-modifying antirheumatic drug (DMARD) therapy: stable dose for at least 3 months before enrollment, and the dose will not be increased during the study. 8. Women of childbearing potential must use an effective contraceptive method and have a negative pregnancy test before study initiation.
Exclusion criteria
Exclusion criteria: 1.Administration of intra-articular, peri-articular, intramuscular, or intravenous glucocorticoids, or use of narcotic analgesics within 4 weeks prior to dosing, except for analgesics permitted during the study (codeine and tramadol). 2.Prior treatment with any of the following agents: Anakinra within 1 week before enrollment; Tofacitinib, baricitinib, upadacitinib, deucravacitinib, abrocitinib, ruxolitinib within 2 weeks before enrollment; Tocilizumab, dapsone, mycophenolate mofetil within 3 weeks before enrollment; Rilonacept, etanercept, thalidomide, cyclosporine, growth hormone within 4 weeks before enrollment; Adalimumab or intravenous immunoglobulin (ivIg) within 8 weeks before enrollment; Golimumab within 10 weeks before enrollment; Infliximab, 6-mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil within 12 weeks before enrollment; Leflunomide within 12 weeks before enrollment, except for subjects who discontinued leflunomide for 4 weeks and underwent accelerated elimination (e.g., oral cholestyramine or activated charcoal), with appropriate medical records provided; Rituximab within 26 weeks before enrollment; Participation in any other clinical trial, or use of other biological agents, within 4 weeks or 5 elimination half-lives before enrollment (whichever is longer). 3.Immunocompromised patients; positive results for hepatitis B surface antigen (HBsAg), hepatitis C antibody, and/or human immunodeficiency virus (HIV) antibody. 4.Recurrent or active infection (bacterial, fungal, or viral) within 4 weeks; history of recurrent invasive fungal infection; 5.Positive T-Spot assay for tuberculosis during screening.Subjects may be included only if there is no history of latent or active tuberculosis before screening, no signs or symptoms suggestive of active tuberculosis, and no recent close contact with patients with active tuberculosis. 6.Abnormal liver function: total bilirubin (TBL) > 1.5 × upper limit of normal (ULN); alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >= 3 × ULN. 7.Serum creatinine concentration > 1.5 mg/dL; eGFR <= 29 mL/min/1.73 m². 8.Abnormal complete blood count: hemoglobin (Hb) <= 100 g/L, neutrophils <= 2.5 × 10?/L, platelets <= 100 × 10?/L. 9.Patients diagnosed with other malignant tumors (except cured cervical cancer, in situ skin cancer, or other surgically cured tumors with no recurrence for at least 5 years). 10.Any patient who has undergone organ transplantation or is scheduled to undergo organ transplantation. 11.Presence of any other severe underlying disease including but not limited to severe cardiovascular, pulmonary, or other systemic disease, which in the investigator’s judgment may interfere with the subject’s ability to participate in the study, undergo treatment and follow-up, affect subject compliance, or potentially lead to complications related to the study drug. 12.History of pericarditis, myocarditis, bacterial heart valve disease, or endocarditis within 6 months prior to screening. 13.History of interstitial lung disease, pulmonary fibrosis, pulmonary alveolar proteinosis, or pulmonary hypertension;asthma requiring parenteral glucocorticoid therapy within 6 months prior to screening;atopic dermatitis requiring pharmacologic intervention within 6 months prior to screening. 14.History of multiple sclerosis or other demyelinating diseases, or Felty’s syndrome;history of severe metabolic disease, hematological disease, recurrent chronic infection, etc., who in th
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| remission rate by week 2; | — |
Countries
China
Contacts
Public ContactMin Shen
Peking Union Medical College Hospital
Outcome results
None listed