drugset / Trial / NCT06660329
Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2024-10-01
- Primary completion
- 2027-10-01
- Completion
- 2028-10-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Tofacitinib | Small molecule | 2 mg | — |
| Subject | Tofacitinib | Small molecule | 2.5 mg | — |
| Subject | Tofacitinib | Small molecule | 3 mg | — |
| Subject | Tofacitinib | Small molecule | 3.5 mg | — |
| Subject | Tofacitinib | Small molecule | 4 mg | — |
| Subject | Tofacitinib | Small molecule | 5 mg | — |