Clinical trial NCT03923140: A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)
Official title: Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Cryopyrin-Associated Periodic Syndromes
Keywords: Cryopyrin-Associated Periodic Syndromes
Tranilast
Efficacy
Safety
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2019-04-22
Enrollment: 71
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: a single-arm prospective cohort study
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility 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

Primary outcomes:
measure: Changes in Auto-Inflammatory Diseases Activity Index score after 6-month treatment over baseline
timeFrame: The previous 1 month before treatment and the 6th month after treatment
Secondary outcomes:
measure: Changes in Auto-Inflammatory Diseases Activity Index score at the 1st and 3rd month over baseline
timeFrame: The previous 1 month before treatment and the 1st and 3rd month after treatment
measure: Changes in inflammatory markers, including C-reactin protein, erythrocyte sedimentation rate, serum amyloid protein, interleukin-1β and interleukin-18， at 1, 3 and 6 months over baseline
timeFrame: Baseline and at 1, 3 and 6 months after treatment
measure: Changes in physician global assessment of disease activity on a 0-10 visual analog scale (VAS) at 1, 3 and 6 months over baseline
timeFrame: Baseline and 1, 3 and 6 months after treatment
measure: 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
timeFrame: Baseline and 1, 3 and 6 months after treatment
measure: Changes in CSF white blood cell count for CINCA patients
timeFrame: Baseline and 6 months after treatment.
measure: Changes in MRI of the brain and inner ear for CINCA patients
timeFrame: Baseline and 6 months after treatment.
measure: Changes in audiology data for CINCA patients
timeFrame: Baseline and 6 months after treatment.
measure: Number of participants with adverse effect
timeFrame: Up to 6 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Peking Union Medical College Hospital
name: Hongmei Song
role: PRINCIPAL_INVESTIGATOR
References: