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

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase

Enrolling

Phase 2

Conditions

Cryopyrin-Associated Periodic Syndromes

Treatments

Drug: Tranilast

Study type

Interventional

Funder types

Other

Identifiers

NCT03923140

ZS-1921

Details and patient eligibility

About

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.

Full description

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.

Enrollment

71 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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