Blau SyndromePhase 4NCT06660329
Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome
A Phase 4 study testing Janus Kinase Inhibitor in people with Blau Syndrome, 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 Tofacitinib in children with Blau syndrome (BS). The investigators would analyze the rate of remission or low disease activity after treatment as well as changes in inflammatory markers, patients' and physician's global assessment of disease… Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Blau Syndrome
- What is being tested?
- Janus Kinase Inhibitor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2024to 1 Oct 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients who have pathogenic mutation(s) in NOD2 gene;
- Patients who have clinical manifestations such as granulomatous dermatitis, arthritis, uveitis, vasculitis, interstitial lung disease and so on;
- Clinical remission was not achieved after ≥12 weeks of treatment with at least one immunosuppressant or biologics.
Who cannot join
- Patients will not be included if meets any of the following criteria:
- Being treated with IL-1 inhibitor, or other biological agents;
- Pregnant and lactating women;
- Serious organ function failure, expected life time less than 6 months.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Tofacitinib in Patients with Refractory Blau Syndrome: a Prospective Cohort Study
This is a prospective cohort study to observe the efficacy and safety of Tofacitinib in children with Blau syndrome (BS). The investigators would analyze the rate of remission or low disease activity 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 Tofacitinib.
- Conditions
- Blau Syndrome
- Being tested
- Janus Kinase Inhibitor
- Keywords
- Blau syndrome, Janus kinase inhibitors
- Medical subject headings
- Blau syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Janus Kinase InhibitorDrug
Tofacitinib is used according to weight: 5~<7kg，2mg；7~<10kg，2.5mg；10~<15kg，3mg；15~<25kg，3.5mg；25~<40kg，4mg；≥40kg，5mg. All is twice a day.
| Study group | Role | What they receive |
|---|---|---|
| Janus kinase inhibitors | Experimental | Tofacitinib is used according to weight: 5~<7kg，2mg；7~<10kg，2.5mg；10~<15kg，3mg；15~<25kg，3.5mg；25~<40kg，4mg；≥40kg，5mg. All is twice a day. Receives: Drug: Janus Kinase Inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
rate of remission or low disease activity
Measured: From enrollment to the end of treatment at 6 months
Secondary outcomes (5)
RCB(Response in Chinese children with Blau syndrome) 30, 50, 70 response rates
Measured: From enrollment to the end of treatment at 3, 6, 9, 12 months
Changes in inflammatory markers (including erythrocyte sedimentation rate, C reactive protein), cytokines (including IL-1β, IL-6, IL-17, IL-18, TNFα, IFN γ) and expression of type I interferon-stimulated genes over baseline
Changes are standardized as the ratio of the numerical difference before and after treatment to the baseline value
Measured: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Proportion of recurrent uveitis
Measured: From enrollment to the end of treatment at 12 months
Incidence of new organ involvement
Measured: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Number of participants with adverse effect
Measured: From enrollment to the end of treatment at 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2024
- Study start1 Oct 2024
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Blau SyndromeStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06660329
- Sponsor's protocol ID
- K6581
- Status last verified
- Oct 2024
- First submitted
- 22 Oct 2024
- Last updated
- 28 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Tofacitinib in Patients with Refractory Blau Syndrome: a Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT06660329
- Acronym / sponsor ID
- K6581
- Conditions
- Blau Syndrome
- Keywords
- Blau syndrome, Janus kinase inhibitors
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Oct 2028 (estimated)
- First posted
- 28 Oct 2024 (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 Tofacitinib in children with Blau syndrome (BS). The investigators would analyze the rate of remission or low disease activity 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 Tofacitinib.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Janus kinase inhibitors | Experimental | Drug: Janus Kinase InhibitorTofacitinib is used according to weight: 5~<7kg，2mg；7~<10kg，2.5mg；10~<15kg，3mg；15~<25kg，3.5mg；25~<40kg，4mg；≥40kg，5mg. All is twice a day. |
Who may qualify
- Ages
- 0 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Patients who have pathogenic mutation(s) in NOD2 gene;
- Patients who have clinical manifestations such as granulomatous dermatitis, arthritis, uveitis, vasculitis, interstitial lung disease and so on;
- Clinical remission was not achieved after ≥12 weeks of treatment with at least one immunosuppressant or biologics.
Key exclusion criteria (4 listed)
- Being treated with IL-1 inhibitor, or other biological agents;
- Pregnant and lactating women;
- Serious organ function failure, expected life time less than 6 months.
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 |
|---|---|---|
| Janus Kinase InhibitorTofacitinib is used according to weight: 5~<7kg，2mg；7~<10kg，2.5mg；10~<15kg，3mg；15~<25kg，3.5mg；25~<40kg，4mg；≥40kg，5mg. All is twice a day. | Drug | Janus kinase inhibitors |
Primary outcome
rate of remission or low disease activity
Time frame: From enrollment to the end of treatment at 6 months
5 secondary outcomes
RCB(Response in Chinese children with Blau syndrome) 30, 50, 70 response rates
Time frame: From enrollment to the end of treatment at 3, 6, 9, 12 months
Changes in inflammatory markers (including erythrocyte sedimentation rate, C reactive protein), cytokines (including IL-1β, IL-6, IL-17, IL-18, TNFα, IFN γ) and expression of type I interferon-stimulated genes over baseline
Time frame: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Proportion of recurrent uveitis
Time frame: From enrollment to the end of treatment at 12 months
Incidence of new organ involvement
Time frame: From enrollment to the end of treatment at 1,3, 6, 9, 12 months
Number of participants with adverse effect
Time frame: From enrollment to the end of treatment at 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2024.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Janus Kinase Inhibitor in people with Blau Syndrome, 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 3 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: enrolling by invitation, as of the sponsor's last update on 28 Oct 2024. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
rate of remission or low disease activity (time frame: From enrollment to the end of treatment at 6 months). 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 28 Oct 2024.