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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

Tracking Information
First Submitted Date  ICMJE April 14, 2019
First Posted Date  ICMJE April 22, 2019
Last Update Posted Date June 4, 2019
Actual Study Start Date  ICMJE May 23, 2019
Estimated Primary Completion Date April 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 19, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03923140 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 19, 2019)
  • 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.
  • 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.
  • 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).
  • 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).
  • 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.
  • 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.
  • 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.
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)
Official Title  ICMJE Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
a single-arm prospective cohort study
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cryopyrin-Associated Periodic Syndromes
Intervention  ICMJE 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
Study Arms  ICMJE 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
Intervention: Drug: Tranilast
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 19, 2019)
71
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2024
Estimated Primary Completion Date April 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Hongmei Song, Doctor +86-10-69156271 songhm1021@hotmail.com
Contact: Linqing Zhong +86-13011825185 zhonglinqing_pumch@126.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03923140
Other Study ID Numbers  ICMJE ZS-1921
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Hongmei Song, Peking Union Medical College Hospital
Study Sponsor  ICMJE Peking Union Medical College Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Hongmei Song Peking Union Medical College Hospital
PRS Account Peking Union Medical College Hospital
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP