Teprotumumab N01 for adults with thyroid eye disease

Evaluating the Efficacy and Safety of Teprotumumab N01 in Patients With Thyroid Eye Disease by FAPI PET/CT and 5.0T-MRI

This will try Teprotumumab N01 in adults with recent-onset thyroid eye disease to see if it reduces eye inflammation and improves vision and quality of life.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Peking University Third Hospital (other)
• Drugs / interventions: teprotumumab
• Locations: 1 site (Beijing)
• Trial ID: NCT07423013 on ClinicalTrials.gov

What this trial studies

This is a prospective, single-arm study enrolling adults with thyroid eye disease of less than nine months' duration who will receive Teprotumumab N01 and serve as their own baseline control. Clinical and ophthalmic measures, visual function tests, and quality-of-life questionnaires will be collected before and after treatment to measure change. Advanced imaging with [18F]AlF-NOTA-FAPI-04 PET/CT and 5.0-T high-resolution MRI will be used to quantify orbital tissue changes. Safety will be tracked through adverse event reporting and laboratory tests, with the primary efficacy endpoint being the overall response rate at Week 24.

Who should consider this trial

Good fit: Adults aged 18–80 years, weighing 45–100 kg, with a clinical diagnosis of TED of under nine months who can receive teprotumumab N01 and attend scheduled visits are the ideal candidates.

Not a fit: Patients with long-standing disease, poorly controlled thyroid function, recent radioactive iodine therapy, thyroid-related optic neuropathy, or persistent corneal ulcers are unlikely to benefit from this intervention.

Why it matters

Potential benefit: If successful, Teprotumumab N01 could reduce orbital inflammation, improve vision and symptoms, and provide an effective treatment option for recent-onset TED.

How similar studies have performed: Prior randomized trials of teprotumumab have demonstrated clinical benefit in thyroid eye disease, while this N01 formulation and the focused use of advanced PET/MRI represent newer real-world and imaging-focused data.

Eligibility criteria

Inclusion Criteria:

1. Able to comply with the study procedures and voluntarily sign the written informed consent form;
2. Male or female subjects aged 18-80 years (inclusive) at screening;
3. Body weight between 45 and 100 kg (inclusive);
4. Meet internationally recognized diagnostic criteria for TED who are receiving teprotumumab N01 treatment；
5. Diagnosed with TED at both the screening and baseline visits;
6. Disease duration of less than 9 months

Exclusion Criteria:

1. Poorly controlled thyroid function, defined as FT3 or FT4 deviating by more than 50% from the normal reference range;
2. Receipt of radioactive iodine therapy within 3 months prior to screening;
3. Thyroid dysfunction-related optic neuropathy, defined as any of the following occurring within the past 6 months due to optic nerve involvement: a decrease in best-corrected visual acuity (BCVA) of ≥2 lines, new visual field defects, or secondary color vision impairment;
4. Corneal ulcer without improvement after treatment, as judged by the investigator;
5. A decrease in CAS score of ≥2 points at baseline compared with screening;
6. Prior treatment at any time before screening with monoclonal antibodies, including but not limited to anti-CD20 antibodies, anti-interleukin-6 antibodies, or anti-IGF-1R antibodies;
7. Prior orbital radiotherapy for TED at any time before screening;
8. Prior use at any time before screening of oral, injectable, topical, or inhaled glucocorticoids at a cumulative dose ≥1 g methylprednisolone equivalent;
9. Receipt within 3 months prior to screening of oral or intravenous glucocorticoids (\<1 g methylprednisolone equivalent), or peribulbar or periocular glucocorticoid injections for TED;
10. Use of any other immunosuppressive agents orally or intravenously within 3 months prior to screening;
11. Vaccination within 1 month prior to screening;
12. Hemoglobin \< 8.5 g/dL, platelet count \< 100 × 10³/µL, white blood cell count \< 3 × 10⁹/L, absolute neutrophil count (ANC) \< 2 × 10⁹/L, or absolute lymphocyte count \< 5 × 10⁸/L;
13. Acute or chronic, active or latent, recurrent bacterial, viral, fungal, or other infections, including but not limited to tuberculosis (positive T-SPOT or imaging findings), hepatitis B (HBsAg or HBcAb positive), hepatitis C (anti-HCV or HCV RNA positive), syphilis, herpes simplex, or herpes zoster;
14. History of inflammatory bowel disease, gastrointestinal ulcer or diverticulitis, Cushing's disease, osteoporosis, or psychiatric disorders;
15. History of immunodeficiency, including HIV infection or AIDS, other acquired or congenital immunodeficiency disorders, or organ transplantation;
16. History of autoimmune diseases, such as systemic lupus erythematosus, rheumatoid arthritis, or Sjögren's syndrome;
17. History or current presence of malignancy (except for completely resected skin squamous cell carcinoma, basal cell carcinoma, or localized cervical carcinoma in situ without evidence of metastasis);
18. Severe cardiovascular or cerebrovascular disease or related treatment history, including but not limited to stroke, transient ischemic attack, acute myocardial infarction, unstable angina, arrhythmia, heart failure, coronary artery bypass grafting, or percutaneous coronary intervention;
19. Severe hepatic or renal insufficiency, defined as liver disease or abnormal liver function with ALT or AST ≥ 1.5 × the upper limit of normal, estimated glomerular filtration rate \< 30 mL/min/1.73 m², or serum creatinine ≥ the upper limit of normal;
20. Poorly controlled diabetes mellitus, defined as fasting blood glucose (FBG) ≥ 7.0 mmol/L or HbA1c ≥ 9.0% at screening, or initiation of new antidiabetic medication (oral or injectable) or a change in the dose of current antidiabetic medication by \> 10% within 2 months prior to screening;
21. Poorly controlled hypertension, defined as systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg, or adjustment of antihypertensive medication (dose or drug class) within 1 month prior to screening;
22. Presence of uncontrolled disease conditions, including but not limited to asthma, psoriasis, or inflammatory bowel disease requiring glucocorticoid treatment at disease onset;
23. History of hypersensitivity or allergy to other monoclonal antibodies;
24. Alcohol, tobacco, drug, or chemical substance abuse; Alcohol abuse: weekly alcohol intake \> 21 units for men or \> 14 units for women (1 unit = 360 mL beer, or 150 mL wine, or 45 mL distilled spirits/Chinese liquor); Tobacco abuse: smoking index (number of cigarettes per day × years of smoking) \> 400;
25. Pregnant or breastfeeding female subjects, or male or female subjects planning pregnancy during the study or within 3 months after study completion, or unwilling to use effective contraception;
26. Participation in another interventional clinical trial within 3 months prior to screening (for investigational drugs, within 5 half-lives, whichever is longer; vitamins and minerals excluded), or intention to participate in another clinical trial during the study;
27. Any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in this clinical trial.

Where this trial is running

Beijing

• Peking University Third Hospital — Beijing, China (RECRUITING)

Study contacts

• Study coordinator: Yi Wang

Conditions: Thyroid Eye Disease