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
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