Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome

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.

NCT Number	NCT06660329
Lead Sponsor	Peking Union Medical College Hospital
Conditions	Blau Syndrome
Enrollment	30 participants
Start Date	2024-10-01
Primary Completion	2027-10-01 (estimated)
Study Completion	2028-10-01 (estimated)
Updated on ClinicalTrials.gov	2024-10-28