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

• Status: Recruiting
• Trial end date: 2024-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Peking Union Medical College Hospital
• Treatments: Tranilast

Criteria

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