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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03923140
• Date of registration: 14/04/2019
• Prospective Registration: Yes
• Primary sponsor: Peking Union Medical College Hospital
• Public title: A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)
• Scientific title: Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
• Date of first enrolment: May 23, 2019
• Target sample size: 71
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03923140
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

China

Contacts

• Name: Hongmei Song, Doctor

• Name: Hongmei Song, Doctor

• Name: Hongmei Song
• Affiliation: Peking Union Medical College Hospital

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cryopyrin-Associated Periodic Syndromes

Intervention(s)

Drug: Tranilast

Primary Outcome(s)

Changes in Auto-Inflammatory Diseases Activity Index score after 6-month treatment over baseline [Time Frame: The previous 1 month before treatment and the 6th month after treatment]

Secondary Outcome(s)

Changes in MRI of the brain and inner ear for CINCA patients [Time Frame: Baseline and 6 months after treatment.]

Changes in parent/patient global assessment of well-being on a 0-10 visual analogue score (VAS) at 1, 3 and 6 months over baseline [Time Frame: Baseline and 1, 3 and 6 months after treatment]

Number of participants with adverse effect [Time Frame: Up to 6 months]

Changes in Auto-Inflammatory Diseases Activity Index score at the 1st and 3rd month over baseline [Time Frame: The previous 1 month before treatment and the 1st and 3rd month after treatment]

Changes in inflammatory markers, including C-reactin protein, erythrocyte sedimentation rate, serum amyloid protein, interleukin-1ß and interleukin-18, at 1, 3 and 6 months over baseline [Time Frame: Baseline and at 1, 3 and 6 months after treatment]

Changes in physician global assessment of disease activity on a 0-10 visual analog scale (VAS) at 1, 3 and 6 months over baseline [Time Frame: Baseline and 1, 3 and 6 months after treatment]

Changes in CSF white blood cell count for CINCA patients [Time Frame: Baseline and 6 months after treatment.]

Changes in audiology data for CINCA patients [Time Frame: Baseline and 6 months after treatment.]

Secondary ID(s)

ZS-1921

Source(s) of Monetary Support

Please refer to primary and secondary sponsors