NCT06660329

Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome

Efficacy and Safety of Tofacitinib in Patients with Refractory Blau Syndrome: a Prospective Cohort Study

• Status: Enrolling By Invitation
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 0 Years – 18 Years
• Healthy volunteers: Not accepted

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 activity to determine the efficacy and safety of Tofacitinib.

Conditions

• Blau Syndrome

Interventions

Type	Name	Description
DRUG	Janus Kinase Inhibitor	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.

Timeline

• Start date: 2024-10-01
• Primary completion: 2027-10-01
• Completion: 2028-10-01
• First posted: 2024-10-28
• Last updated: 2024-10-28

Locations

1 site across 1 country: China