Role of the suppressor of cytokine signaling-3 in the pathogenesis of Graves' orbitopathy.
Wonjin Kim, Mi-Kyoung Seo, Yong Joon Kim...
https://pubmed.ncbi.nlm.nih.gov/40104138Actively Recruiting
Phase 2
Age: 18Years - 75Years
All Genders
ID07547930
Tofacitinib Treatment for Glucocorticoid-Resistant Moderate-to-Severe Active Thyroid Eye Disease: A Single-Arm Exploratory Study
Led by The First Affiliated Hospital of Xiamen University · Updated on 2026-04-29
8
Participants Needed
1
Research Sites
N/A
Total Duration
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AI-Summary
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 drug's 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.
CONDITIONS
Brief Title
Tofacitinib for Glucocorticoid-Resistant Moderate-to-Severe Thyroid Eye Disease
Who Can Participate
Age: 18Years - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 to 75 years
- Clinical diagnosis of Graves' orbitopathy (GO) according to EUGOGO criteria
- Moderate-to-severe GO impacting daily life but not sight-threatening
- Active disease with Clinical Activity Score (CAS) of 3 or higher
- Steroid-resistant (no improvement after at least 3g intravenous methylprednisolone) or steroid-intolerant (contraindications or severe side effects)
- Euthyroid or mild thyroid dysfunction controlled with stable medication for at least 4 weeks
- Women of childbearing potential must use effective contraception during the study and for 4 weeks after
- Able and willing to provide informed consent and comply with study procedures
You will not qualify if you...
- Sight-threatening disease such as Dysthyroid Optic Neuropathy or severe corneal breakdown requiring surgery
- Chronic or inactive disease with Clinical Activity Score below 3
- Prior orbital treatments: radiotherapy, surgical decompression, or eye muscle/eyelid surgery within 3 months
- Use of other biologic immunomodulators within 3 months prior to baseline
- Active infections including tuberculosis, hepatitis B or C, HIV, or recent severe infections
- History of blood clots or coagulation disorders
- History of malignancy within past 5 years, except certain skin or cervical cancers
- Significant blood or liver test abnormalities or reduced kidney function as defined by study thresholds
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 24 weeks
Participants receive oral Tofacitinib (5 mg twice daily) to target the inflammatory and tissue remodeling processes in thyroid eye disease.
Visits at Baseline, Week 4, Week 12, and Week 24
Follow-up
Duration - 12 weeks
Participants are monitored for disease relapse, safety, and durability of response after stopping Tofacitinib.
1 visit at Week 36
Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Xiamen University
Xiamen, Fujian, China, 361003
Actively Recruiting
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Research Team
F
Fangsen Xiao, MD
L
Liyin Wang, MM
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Published Research Related To This Trial
Tofacitinib for Refractory Thyroid Eye Disease.
Hossein Ghahvehchian, Bahram Eshraghi
https://pubmed.ncbi.nlm.nih.gov/41165674Thyroid eye disease (Graves' orbitopathy): clinical presentation, epidemiology, pathogenesis, and management.
Wilmar M Wiersinga, Anja K Eckstein, Miloš Žarković
https://pubmed.ncbi.nlm.nih.gov/40324443The 2021 European Group on Graves' orbitopathy (EUGOGO) clinical practice guidelines for the medical management of Graves' orbitopathy.
L Bartalena, G J Kahaly, L Baldeschi...
https://pubmed.ncbi.nlm.nih.gov/34297684