Cryopyrin-Associated Periodic SyndromesPhase 2NCT03923140
A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)
A Phase 2 study testing Tranilast in people with Cryopyrin-Associated Periodic Syndromes, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cryopyrin-Associated Periodic Syndromes
- What is being tested?
- Tranilast
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 1 siteChina
- Study dates
- 23 May 2019to Oct 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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;
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- Musculoskeletal symptoms (arthralgia/arthritis/myalgia);
- Chronic aseptic meningitis;
- Skeletal abnormalities (epiphyseal overgrowth/frontal bossing).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Hongmei Song, Doctor, +86-10-69156271, songhm1021@hotmail.com
Linqing Zhong, +86-13011825185, zhonglinqing_pumch@126.com
About this study
Official title: Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
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.
Read the 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.
- Conditions
- Cryopyrin-Associated Periodic Syndromes
- Being tested
- Tranilast
- Keywords
- Cryopyrin-Associated Periodic Syndromes, Tranilast, Efficacy, Safety
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TranilastDrug
Also called Rizaben
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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Tranilast |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
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).
Measured: 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
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).
Measured: Baseline and 1, 3 and 6 months after treatment
Changes in CSF white blood cell count for CINCA patients
For CINCA patients, Lumbar punctures (LPs) were performed at baseline and 6 months after treatment.
Measured: Baseline and 6 months after treatment.
Changes in MRI of the brain and inner ear for CINCA patients
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.
Measured: Baseline and 6 months after treatment.
Changes in audiology data for CINCA patients
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.
Measured: Baseline and 6 months after treatment.
Number of participants with adverse effect
Treatment-related adverse effect, including abnormal liver function, hematuria and decreased white blood cells
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2019
- Study start23 May 2019
- Primary completionApr 2024 (estimated)
- Study completionOct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cryopyrin-Associated Periodic SyndromesStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- a single-arm prospective cohort study
- Masking
- None (open label)
- Enrolment
- 71 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hongmei Song, Peking Union Medical College Hospital
- Investigators
- Hongmei Song, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03923140
- Sponsor's protocol ID
- ZS-1921
- Status last verified
- Jun 2019
- First submitted
- 14 Apr 2019
- Last updated
- 4 Jun 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Tranilast in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS): A Single-Arm Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT03923140
- Acronym / sponsor ID
- ZS-1921
- Conditions
- Cryopyrin-Associated Periodic Syndromes
- Keywords
- Cryopyrin-Associated Periodic Syndromes, Tranilast, Efficacy, Safety
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 71 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 23 May 2019 (actual)
- Primary completion
- Apr 2024 (estimated)
- Study completion
- Oct 2024 (estimated)
- First posted
- 22 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- a single-arm prospective cohort study
| Arm | Type | Receives |
|---|---|---|
| Tranilast | Experimental | Drug: Tranilast5mg/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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- All patients must meet the following diagnostic criteria of CAPS and have pathogenic mutation(s) in NLRP3 gene.
Key exclusion criteria (4 listed)
- Patients will not be included if meets any of the following criteria:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TranilastAlso: Rizaben5mg/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 |
Primary outcome
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.
8 secondary outcomes
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
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
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
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
Changes in CSF white blood cell count for CINCA patients
Time frame: 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.
Changes in audiology data for CINCA patients
Time frame: Baseline and 6 months after treatment.
Number of participants with adverse effect
Time frame: Up to 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hongmei Song principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tranilast in people with Cryopyrin-Associated Periodic Syndromes, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hongmei Song, Doctor (+86-10-69156271, songhm1021@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2019.