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
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Condition or disease | Intervention/treatment | Phase |
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Cryopyrin-Associated Periodic Syndromes | Drug: Tranilast | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 71 participants |
Allocation: | N/A |
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 |

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
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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 |
- 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.
- 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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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)
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≥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).
Exclusion Criteria:
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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

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
Contact: Hongmei Song, Doctor | +86-10-69156271 | songhm1021@hotmail.com | |
Contact: Linqing Zhong | +86-13011825185 | zhonglinqing_pumch@126.com |
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 |
Principal Investigator: | Hongmei Song | Peking Union Medical College Hospital |
Responsible Party: | Hongmei Song, Professor, Peking Union Medical College Hospital |
ClinicalTrials.gov Identifier: | NCT03923140 |
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 |
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 |
Cryopyrin-Associated Periodic Syndromes Tranilast Efficacy Safety |
Cryopyrin-Associated Periodic Syndromes Syndrome 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 |