Cryopyrin-associated periodic syndrome
Conditions
Interventions
Experimental:Firsekibart , 3mg/kg, maximum dose 200mg,
Sponsors
Peking Union Medical College Hospital
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: 1.Male or female patients aged 6 months to less than 18 years; 2.Clinically diagnosed with CAPS; 3.Presence of an NLRP3 pathogenic or likely pathogenic variant consistent with CAPS as confirmed by genetic testing; 4.Active disease during the screening period; 5.The subject’s legal guardian voluntarily signs the written informed consent form; age-appropriate children sign the assent form as required.
Exclusion criteria
Exclusion criteria: 1.Concomitant active severe infection; 2.Known active tuberculosis, latent tuberculosis infection, or a risk of tuberculosis infection that has not been adequately evaluated and managed; active hepatitis B or active HBV DNA replication; active hepatitis C infection; or HIV positivity; 3.History of severe hypersensitivity to Firsekibart or any of its excipients; 4.Use of the following medications before enrollment without meeting the prespecified washout period: • Use of anakinra within 1 week prior to enrollment; • Use of tofacitinib, baricitinib, upadacitinib, deucravacitinib, abrocitinib, or ruxolitinib within 2 weeks prior to enrollment; • Use of tocilizumab, dapsone, or mycophenolate mofetil within 3 weeks prior to enrollment; • Use of rilonacept, etanercept, thalidomide, cyclosporine, or growth hormone within 4 weeks prior to enrollment; • Use of adalimumab or intravenous immunoglobulin within 8 weeks prior to enrollment; • Use of golimumab within 10 weeks prior to enrollment; • Use of infliximab, 5-fluorouracil, azathioprine, cyclophosphamide, or chlorambucil within 12 weeks prior to enrollment; • Use of leflunomide within 12 weeks prior to enrollment, except for subjects who discontinued leflunomide for at least 4 weeks and underwent an accelerated elimination procedure, such as oral cholestyramine or activated charcoal; • Use of rituximab within 26 weeks prior to enrollment; • Participation in any other clinical trial, or use of any other biologic agent, within 4 weeks prior to enrollment or within 5 drug half-lives, whichever is longer. 5.Concomitant systemic inflammatory disease, autoimmune disease, or severe organic disease that may significantly interfere with the evaluation of efficacy or safety; 6.Abnormal liver function, defined as total bilirubin (TBL) >1.5 × the upper limit of normal (ULN), or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >=3 × ULN; 7.Serum creatinine >132.6 µmol/L or eGFR <=29 mL/min/1.73 m^2; 8.Abnormal complete blood count, defined as hemoglobin <=100 g/L, neutrophils <=1.5 × 10^9/L, or platelets <=100 × 10^9/L; 9.Receipt of a live vaccine shortly before screening; 10.Any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Complete response rate at Week 20; | — |
Secondary
| Measure | Time frame |
|---|---|
| Trend in disease activity changes across study visits;Descriptive analysis of efficacy among subjects with different CAPS phenotypes;Change in CRP, ESR, and SAA from baseline at Weeks 8, 16, and 20;Change in AIDAI from baseline at Weeks 8, 16, and 20;Rate of normalization of inflammatory markers at Week 20; | — |
Countries
China
Contacts
Public ContactMa Mingsheng
Peking Union Medical College Hospital
Outcome results
None listed