Evaluating the Efficacy and Safety of Teprotumumab N01 in Patients With Thyroid Eye Disease.

NCT07423013 | Recruiting

• 1 other identifier: Teprotumumab N01-TED
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective study designed to evaluate the efficacy and safety of Teprotumumab N01 in patients with Thyroid Eye Disease (TED). Eligible patients will receive Teprotumumab N01 and will be assessed using clinical and imaging parameters before and after treatment, with each patient serving as their own control. The primary endpoint is the overall response rate at Week 24.

Conditions

Thyroid Eye Disease

Outcome Measures

Primary Outcomes (3)

• To evaluate the effect of teprotumumab N01 on the response rate in patients with TED

  The overall response rate is defined as the proportion of subjects achieving a predefined therapeutic response in the study eye. A response is defined as improvement in ≥2 of the following 5 criteria compared with baseline, with no worsening in any criterion in either eye: 1. Clinical Activity Score (CAS) reduction ≥2 points (7-item CAS: eyelid erythema, eyelid edema, conjunctival injection, chemosis, caruncle swelling, spontaneous retrobulbar pain, pain on eye movement; 1 point each); 2. Proptosis reduction ≥2 mm; 3. Palpebral fissure width (height) reduction ≥2 mm; 4. Diplopia improvement (≥1 grade improvement on the Bahn-Gorman subjective diplopia scale \[0-3\]) or improvement in ocular motility ≥8°; 5. Soft tissue involvement improvement ≥2 grades (based on class 2 NOSPECS grading and EUGOGO color atlas evaluation).

  weeks 24

• To evaluate treatment response using [¹⁸F]AlF-NOTA-FAPI-04 PET/CT in patients with thyroid eye disease

  Response will be assessed using \[¹⁸F\]AlF-NOTA-FAPI-04 PET/CT . These imaging modalities will quantitatively evaluate orbital inflammation, fibroblast activation, and structural changes in the orbit. Imaging-based response will be defined as significant reduction in radiotracer uptake on PET/CT compared with baseline.

  Weeks 24 and 48

• To evaluate treatment response using 5.0-T high-resolution magnetic resonance imaging (MRI) in patients with thyroid eye disease

  Response will be assessed using 5.0-T high-resolution magnetic resonance imaging (MRI). These imaging modalities will quantitatively evaluate orbital inflammation, fibroblast activation, and structural changes in the orbit. Imaging-based response will be defined as improvement in anatomical and inflammatory parameters on MRI compared with baseline.

  Weeks 24 and 48

Secondary Outcomes (13)

• To evaluate the effect of teprotumumab N01 on the response rate in patients with TED

  weeks 48

• To evaluate the recurrence rate of TED after discontinuation of teprotumumab N01.

  weeks 48

• Change in Clinical Activity Score (CAS)

  Weeks 24 and 48

• Improvement in Proptosis

  Weeks 24 and 48

• Improvement in Palpebral Fissure Width (Height)

  Weeks 24 and 48

Study Arms (1)

Experimental arm

EXPERIMENTAL

Patients with TED who are receiving teprotumumab N01 treatment will be recruited in the study

Diagnostic Test: [18F]AlF-NOTA-FAPI-04PET/CT; Diagnostic Test: 5.0-T high-resolution MRI

Interventions

[18F]AlF-NOTA-FAPI-04PET/CT DIAGNOSTIC_TEST

\[¹⁸F\]AlF-NOTA-FAPI-04 PET/CT is used as a molecular imaging intervention in Thyroid Eye Disease to noninvasively assess fibroblast activation protein expression in orbital tissues. It enables evaluation of disease activity, orbital involvement, and treatment response by providing quantitative functional imaging beyond conventional anatomical modalities.

Experimental arm

5.0-T high-resolution MRI DIAGNOSTIC_TEST

5.0-T high-resolution MRI is used as an imaging intervention in Thyroid Eye Disease to provide detailed anatomical visualization of the orbit, including extraocular muscles, orbital fat, optic nerve, and soft tissues. It allows precise assessment of disease extent, structural changes, and treatment-related morphological responses.

Experimental arm

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Poorly controlled thyroid function, defined as FT3 or FT4 deviating by more than 50% from the normal reference range;
• Receipt of radioactive iodine therapy within 3 months prior to screening;
• 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;
• Corneal ulcer without improvement after treatment, as judged by the investigator;
• A decrease in CAS score of ≥2 points at baseline compared with screening;
• 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;
• Prior orbital radiotherapy for TED at any time before screening;
• Prior use at any time before screening of oral, injectable, topical, or inhaled glucocorticoids at a cumulative dose ≥1 g methylprednisolone equivalent;
• Receipt within 3 months prior to screening of oral or intravenous glucocorticoids (\<1 g methylprednisolone equivalent), or peribulbar or periocular glucocorticoid injections for TED;
• Use of any other immunosuppressive agents orally or intravenously within 3 months prior to screening;
• Vaccination within 1 month prior to screening;
• 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;
• 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;
• History of inflammatory bowel disease, gastrointestinal ulcer or diverticulitis, Cushing's disease, osteoporosis, or psychiatric disorders;
• History of immunodeficiency, including HIV infection or AIDS, other acquired or congenital immunodeficiency disorders, or organ transplantation;

Sponsors & Collaborators

• Peking University Third Hospital: lead
• Innovent Biologics (Suzhou) Co. Ltd.: collaborator

Study Sites (1)

Peking University Third Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Graves Ophthalmopathy

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Graves Disease; Exophthalmos; Orbital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Goiter; Thyroid Diseases; Endocrine System Diseases; Hyperthyroidism; Autoimmune Diseases; Immune System Diseases

Study Officials

• Yi Wang

  Peking University Third Hospital

  STUDY CHAIR

Central Study Contacts

Yi Wang

CONTACT

86-13439150508; wangyieye@126.com

Lingge Suo

CONTACT

86-010-82264935; suolingge_1019@126.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2025
• First Posted: February 20, 2026
• Study Start: November 11, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): May 31, 2027
• Last Updated: February 20, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)