Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome

NCT ID: NCT06660329

Last Updated: 2024-10-28

Basic Information

• Recruitment Status: ENROLLING_BY_INVITATION
• Clinical Phase: PHASE4
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-10-01
• Study Completion Date: 2028-10-01

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Janus kinase inhibitors

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.

Group Type: EXPERIMENTAL

Janus Kinase Inhibitor

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

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.

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Hongmei Song

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking Union Medical College Hospital

Beijing, , China

Countries

China

Other Identifiers

K6581

Identifier Type: -

Identifier Source: org_study_id