A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)

Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study

This is a prospective cohort study to observe the efficacy and safety of tranilast in CAPS patients. The investigators would analyze the changes in Auto-Inflammatory Diseases Activity Index (AIDAI) before and after treatment as well as changes in inflammatory markers, patients' and physician's global assessment of disease activity to determine the efficacy and safety of tranilast.

Seventy-one patients with CAPS will be recruited. After signing the informed consent, they will be administrated with tranilast (For juvenile patients, 5mg/kg.d with a maximum dose of 0.3g per day; For adult patients, the dose is 0.1g each time, three times a day). These patients will be followed up for 6 months. AIDAI is recorded by patients' or their parents one month before the start of treatment, and at the 1st, 3rd and 6th month after the treatment. Inflammatory markers, and patients' and physician's global assessment of disease activity will be assessed during the 1st, 3rd and 6th month follow-up. Side effects will be monitored and recorded as well. Experimental data before and after the administration of tranilast will be analyzed and be statistically processed, to figure out whether tranilast is effective and safe for CAPS patients.

Inclusion Criteria:

• All patients must meet the following diagnostic criteria of CAPS and have pathogenic mutation(s) in NLRP3 gene.

  1. Raised inflammatory markers (CRP/SAA) (mandatory criteria)

  2. ≥2 of 6 CAPS typical signs/symptoms:

     1. Urticaria-like rash;
     2. Cold/stress triggered episodes;
     3. Sensorineural hearing loss;
     4. Musculoskeletal symptoms (arthralgia/arthritis/myalgia);
     5. Chronic aseptic meningitis;
     6. Skeletal abnormalities (epiphyseal overgrowth/frontal bossing).

Exclusion Criteria:

• Patients will not be included if meets any of the following criteria:

  1. Being treated with IL-1 inhibitor, other biological agents and immunosuppressants
  2. Pregnant and lactating women
  3. Serious organ function failure, expected life time less than 6 months