Study of Oral Tofacitinib for Adults With Moderate-to-Severe Thyroid Eye Disease Not Responding to Steroids

What this Trial Is About

Thyroid Eye Disease TED, also known as Graves orbitopathy, is an autoimmune condition that causes inflammation and tissue expansion behind the eyes, leading to bulging eyes, double vision, and pain. This trial evaluates the oral medication Tofacitinib, a Janus kinase JAK inhibitor, as a rescue therapy for patients with moderate-to-severe active TED who are resistant or intolerant to standard steroid treatments. The study seeks to assess the safety and effectiveness of Tofacitinib in reducing eye bulging and improving clinical activity. Participants will receive oral Tofacitinib tablets at a dose of 5 mg twice daily for 24 weeks. This is a single-arm, open-label Phase 2 study where all participants receive the treatment. After completing the 24-week treatment period, participants enter a 12-week observation phase without the drug to monitor for disease relapse and safety. The study includes detailed imaging and clinical assessments to understand the drugs effects on inflammation and tissue remodeling. During the study, participants will have clinical evaluations at baseline, weeks 4, 12, 24, and a follow-up at week 36. Assessments include measurement of eye bulging, clinical activity scores, quality of life questionnaires, and monitoring of side effects. Orbital MRI scans will be used to measure changes in muscle and fat volume behind the eyes. Safety monitoring includes blood tests for coagulation and lipid levels. Overall, the study lasts about 36 weeks, including treatment and follow-up periods.

Tofacitinib for Glucocorticoid-Resistant Moderate-to-Severe Thyroid Eye Disease