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

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ClinicalTrials.gov Identifier: NCT03923140
Recruitment Status : Recruiting
First Posted : April 22, 2019
Last Update Posted : June 4, 2019
Sponsor:
Information provided by (Responsible Party):
Hongmei Song, Peking Union Medical College Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Cryopyrin-Associated Periodic Syndromes Drug: Tranilast Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 71 participants
Intervention Model: Single Group Assignment
Intervention Model Description: a single-arm prospective cohort study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
Actual Study Start Date : May 23, 2019
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Tranilast
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
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
Other Name: Rizaben


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
    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.


Secondary Outcome Measures :
  1. 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 ]
    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 1st and 3rd 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.

  2. 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 [ Time Frame: Baseline and at 1, 3 and 6 months after treatment ]
    C-reactin protein, erythrocyte sedimentation rate, serum amyloid protein, interleukin-1β and interleukin-18 are measured at baseline，1, 3 and 6 months after treatment.

  3. 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 ]
    Visual analogue scale (VAS) for overall disease activity were completed by the physician at each visit (Baseline and 1, 3 and 6 months after treatment).

  4. 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 ]
    Visual analogue scale (VAS) for overall disease activity were completed by the parent/patient at each visit (Baseline and 1, 3 and 6 months after treatment).

  5. Changes in CSF white blood cell count for CINCA patients [ Time Frame: Baseline and 6 months after treatment. ]
    For CINCA patients, Lumbar punctures (LPs) were performed at baseline and 6 months after treatment.

  6. Changes in MRI of the brain and inner ear for CINCA patients [ Time Frame: Baseline and 6 months after treatment. ]
    For CINCA patients, MRIs with gadoliniumenhanced fluid-attenuated inversion recovery (FLAIR) sequences of the brain and inner ear were performed and scored at baseline and 6 months after treatment.

  7. Changes in audiology data for CINCA patients [ Time Frame: Baseline and 6 months after treatment. ]
    For CINCA patients, Hearing assessment included audiological evaluations. Outcomes in each ear were categorised as 'stable' or 'worsened', according to a modification of the American Speech and Hearing Association (ASHA) criteria, comparing the results at 6 months after treatment over baseline.

  8. Number of participants with adverse effect [ Time Frame: Up to 6 months ]
    Treatment-related adverse effect, including abnormal liver function, hematuria and decreased white blood cells


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03923140


Contacts
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Contact: Hongmei Song, Doctor +86-10-69156271 songhm1021@hotmail.com
Contact: Linqing Zhong +86-13011825185 zhonglinqing_pumch@126.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Hongmei Song, Doctor    +86-10-69156271    songhm1021@hotmail.com   
Contact: Linqing Zhong    +86-13011825185    zhonglinqing_pumch@126.com   
Principal Investigator: Hongmei Song, Doctor         
Sub-Investigator: Linqing Zhong         
Sponsors and Collaborators
Peking Union Medical College Hospital
Investigators
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Principal Investigator: Hongmei Song Peking Union Medical College Hospital
More Information
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Responsible Party: Hongmei Song, Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03923140     History of Changes
Other Study ID Numbers: ZS-1921
First Posted: April 22, 2019    Key Record Dates
Last Update Posted: June 4, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Hongmei Song, Peking Union Medical College Hospital:
Cryopyrin-Associated Periodic Syndromes
Tranilast
Efficacy
Safety

Additional relevant MeSH terms:
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Syndrome
Cryopyrin-Associated Periodic Syndromes
Disease
Pathologic Processes
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Tranilast
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
 Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Calcium-Regulating Hormones and Agents
Histamine H1 Antagonists
Histamine Antagonists
Histamine Agents
Neurotransmitter Agents
Platelet Aggregation Inhibitors
Anti-Allergic Agents