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

NCT03923140 | Unknown Phase 2

• 1 other identifier: ZS-1921
• Study Type: interventional
• Target: 71
• Locations: 1 country
• Sites: 1

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.

Conditions

Cryopyrin-Associated Periodic Syndromes

Keywords

Cryopyrin-Associated Periodic Syndromes; Tranilast; Efficacy; Safety

Outcome Measures

Primary Outcomes (1)

• Changes in Auto-Inflammatory Diseases Activity Index score after 6-month treatment over baseline

  Patients or their parents completed a 1-month (31 days) prospective diary with 12 yes/no items( Fever ≥38°C, Overall symptoms, Abdominal pain, Nausea/vomiting, Diarrhoea, Headaches, Chest pain, Painful nodes, Arthralgia or myalgia, Swelling of the joints, Eye manifestations, Skin rash) at the previous 1 month before treatment, and the 6th month after treatment . Each item of this diary was dichotomised as no (0)=absence of symptom or yes (1)=presence of symptom. The calculation of the Auto-Inflammatory Diseases Activity Index score is straightforward, consisting of the sum of all 12 items (0-372 in a month of 31 days). Higher values represent higher disease activity.

  The previous 1 month before treatment and the 6th month after treatment

Secondary Outcomes (8)

• Changes in Auto-Inflammatory Diseases Activity Index score at the 1st and 3rd month over baseline

  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-1β and interleukin-18， at 1, 3 and 6 months over baseline

  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

  Baseline and 1, 3 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

  Baseline and 1, 3 and 6 months after treatment

• Changes in CSF white blood cell count for CINCA patients

  Baseline and 6 months after treatment.

Study Arms (1)

Tranilast

EXPERIMENTAL

5mg/kg.d for juvenile patients with a maximum dose of 0.3g per day; 0.1g each time, three times a day for adults patients

Drug: Tranilast

Interventions

Tranilast DRUG

5mg/kg.d for juvenile patients with a maximum dose of 0.3g per day; 0.1g each time, three times a day for adults patients

Also known as: Rizaben

Tranilast

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• All patients must meet the following diagnostic criteria of CAPS and have pathogenic mutation(s) in NLRP3 gene.
• Raised inflammatory markers (CRP/SAA) (mandatory criteria)
• ≥2 of 6 CAPS typical signs/symptoms:
• Urticaria-like rash;
• Cold/stress triggered episodes;
• Sensorineural hearing loss;
• Musculoskeletal symptoms (arthralgia/arthritis/myalgia);
• Chronic aseptic meningitis;
• Skeletal abnormalities (epiphyseal overgrowth/frontal bossing).

You may not qualify if:

• Patients will not be included if meets any of the following criteria:
• Being treated with IL-1 inhibitor, other biological agents and immunosuppressants
• Pregnant and lactating women
• Serious organ function failure, expected life time less than 6 months

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Interventions

tranilast

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Hongmei Song

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hongmei Song, Doctor

CONTACT

+86-10-69156271; songhm1021@hotmail.com

Linqing Zhong

CONTACT

+86-13011825185; zhonglinqing_pumch@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: a single-arm prospective cohort study

Study Record Dates

• First Submitted: April 14, 2019
• First Posted: April 22, 2019
• Study Start: May 23, 2019
• Primary Completion: April 1, 2024
• Study Completion: October 1, 2024
• Last Updated: June 4, 2019

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)