Tofacitinib for Glucocorticoid-Resistant Moderate-to-Severe Thyroid Eye Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorThe First Affiliated Hospital of Xiamen University
About this trial
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 (proptosis), double vision, and pain. Currently, intravenous glucocorticoids (steroids) are the standard first-line treatment. However, approximately 20-30% of patients do not respond to steroids, or cannot tolerate their side effects.
This study aims to evaluate the safety and efficacy of Tofacitinib, an oral medication known as a Janus kinase (JAK) inhibitor, as a rescue therapy for these difficult-to-treat cases. Tofacitinib works by blocking specific signaling pathways (JAK-STAT) that drive inflammation and fibrosis in the eye socket. In this study, patients with moderate-to-severe active TED who are resistant to or intolerant of steroids will receive Tofacitinib tablets (5 mg twice daily) for 24 weeks. The researchers will assess whether the treatment can effectively reduce eye bulging and improve clinical activity scores.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age and Gender: Male or female participants aged 18 to 75 years (inclusive).
Diagnosis: Clinical diagnosis of Graves' orbitopathy (GO) consistent with EUGOGO criteria.
Severity: Moderate-to-severe GO as defined by EUGOGO guidelines (impact on daily life, but not sight-threatening).
Activity: Active disease, defined as a Clinical Activity Score (CAS) >=3 points (on the 7-point scale).
Disqualifiers
1.Sight-Threatening Disease: Presence of Dysthyroid Optic Neuropathy (DON) or severe corneal breakdown requiring immediate surgical intervention.
Trial design
Design model
Single group
Treatments tested in this trial
Tofacitinib
Drug5 mg tablet administered orally twice daily (BID) for a continuous period of 24 weeks.
Treatment groups
8 Participants
are divided into 1 treatment groupGroup A: Tofacitinib Treatment GroupExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Overall Response Rate (ORR)
Percentage of participants classified as responders in the study eye. A responder is defined as a patient who achieves at least one of the following criteria without deterioration in the fellow eye: (1) Reduction in proptosis\>=2 mm; (2) Reduction in Clinical Activity Score (CAS) \>= 2 points (on a 7-point scale).
Time frame
Week12，24Secondary outcomes
1
Change in Proptosis
Mean change in proptosis from baseline in the study eye, measured by Hertel exophthalmometer in millimeters (mm).
Time frame
Baseline, Week 4, 12, 24, and 362
Change in Clinical Activity Score (CAS)
Mean change in Clinical Activity Score (CAS) from baseline. CAS is assessed on a 7-point scale (1 point for each: spontaneous retrobulbar pain, pain on gaze, eyelid erythema, eyelid swelling, conjunctival injection, chemosis, inflammation of caruncle/plica), where higher scores indicate more active inflammation.
Time frame
Baseline, Week 4, 12, 24, and 363
Diplopia Response
ercentage of participants with improvement in diplopia. Improvement is defined as a reduction of at least one grade in the Gorman diplopia score (Grades: absent, intermittent, inconstant, constant)
Time frame
12W,24W4
Change in Graves' Orbitopathy Quality of Life (GO-QOL) Score
Mean change in the Graves' Orbitopathy Quality of Life (GO-QOL) questionnaire score from baseline. The GO-QOL measures two subscales: visual functioning and appearance. For each subscale, the minimum score is 0 and the maximum score is 100. Higher scores indicate a better quality of life.
Time frame
Baseline and Week4，12，24，36Sponsors and contacts
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Source ClinicalTrials.gov