NCT03000439

A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA

EFFICACY, SAFETY, TOLERABILITY AND PHARMACOKINETICS OF TOFACITINIB FOR TREATMENT OF SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (SJIA) WITH ACTIVE SYSTEMIC FEATURES IN CHILDREN AND ADOLESCENT SUBJECTS

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

A randomized withdrawal study in which responders to open-label treatment with tofacitinib will be randomized in a 1:1 ratio to tofacitinib or placebo in a double-blind phase. In the double-blind phase "time to sJIA flare" will be evaluated as primary endpoint and subjects will be discontinued once they experience sJIA flare. An interim analysis for efficacy and futility will be conducted when at least 20 flares have been observed. If either criterion is met, the study will be stopped. If neither criterion is met, the study will continue until the requisite number of flares are observed as determined by the number of flares included in the interim analysis and a statistical penalty for conducting the interim analysis.

Conditions

• Arthritis Juvenile Idiopathic

Interventions

Type	Name	Description
DRUG	In open-label phase: treatment with tofacitinib	Treatment with investigational drug
DRUG	In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio	Treatment with investigational drug or placebo

Timeline

• Start date: 2018-05-10
• Primary completion: 2024-03-27
• Completion: 2024-03-27
• First posted: 2016-12-22
• Last updated: 2025-06-03
• Results posted: 2025-06-03

Locations

52 sites across 19 countries: United States, Argentina, Belgium, Brazil, Canada, China, Costa Rica, Germany, Hungary, India, Israel, Italy, Mexico, Poland, Russia, South Africa, Spain, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study