A Study of Teprotumumab N01 in Subjects With Active Thyroid Eye Disease

NCT07265258 | Not Yet Recruiting Phase 4

• 1 other identifier: CIBI311A303
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multicenter, randomized, open-label, active-controlled Phase IV clinical trial in participants with active thyroid eye disease (TED). Approximately 92 eligible participants will be randomized to the teprotumumab N01 group and the intravenous glucocorticoid (IVGC) group in a 1:1 ratio on Day 1. The randomization stratification factor is diplopia at baseline (Gorman diplopia score ≥1 vs. Gorman diplopia score = 0).

Conditions

Thyroid Eye Disease

Outcome Measures

Primary Outcomes (1)

• The proptosis responder rate of the study eye

  The proptosis response rate of the study eye was defined as the percentage of participants with a ≥ 2 mm reduction in proptosis of the study eye from baseline and without a ≥ 2 mm increase in proptosis of the fellow eye. Proptosis assessment: protrusion of the eye from the orbital rim as measured using a Hertel exophthalmometer.

  Week15

Secondary Outcomes (12)

• Change from baseline in proptosis of the study eye

  Weeks 15, 24 and 48

• The overall response rate of the study eye

  Weeks 15, 24 and 48

• Change from baseline in proptosis of the study eye on MRI imaging

  Week15

• The proptosis response rate of the study eye

  Weeks 24 and 48

• The composite effective rate of the study eye

  Weeks 15, 24 and 48

Study Arms (2)

Intravenous glucocorticoid (IVGC) group

ACTIVE COMPARATOR

Drug: Methylprednisolone

Teprotumumab N01 group

EXPERIMENTAL

Biological: Teprotumumab N01

Interventions

Teprotumumab N01 BIOLOGICAL

A total of 8 intravenous infusions of Teprotumumab N01 injection will be adminsitered: 10 mg/kg on Day1, followed by 20 mg/kg in Week3-21, once every 3 weeks(Q3W).

Teprotumumab N01 group

Methylprednisolone DRUG

A total of 12 intravenous infusions of methylprednisolone will be administered: 500 mg for the first six doses (on Day 1 and weekly throughout Weeks 1-5), and then 250 mg for the following six doses (weekly throughout Weeks 6-11). Based on disease deterioration and treatment response, participants will receive individualized treatment after IVGC therapy.

Intravenous glucocorticoid (IVGC) group

Eligibility Criteria

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

You may qualify if:

• Written informed consent.
• Male or female subject between the ages of 18 and 80 years at screening.
• Weight between 45 kg and 100 kg.
• Moderate-to-severe active TED:
• CAS ≥ 3 in the study eye at screening and baseline;
• Usually associated with at least two of the following: lid retraction ≥ 2 mm, moderate or severe soft tissue involvement, exophthalmos ≥ 3 mm above normal, and/or inconstant or constant diplopia;
• ≤ 12 months since the onset of active TED symptoms according to subjects' chief complaint or medical record at screening;
• Exophthalmos ≥ 16 mm in the study eye at baseline.
• Infertile female participants or fertile female participants with negative blood pregnancy test results during the screening period and agree to take contraceptive measures from screening to 120 days after the last dose; male participants should agree to use contraceptive measures from screening to 120 days after the last dose.

You may not qualify if:

• Participants to be excluded (Participants meeting any of the following criteria will be regarded as ineligible):
• The CAS of the study eye at baseline is reduced by ≥ 2 points compared with that at screening, or the proptosis of the study eye at baseline is reduced by ≥ 2 mm compared with that at screening;
• Participants previously diagnosed with dysthyroid optic neuropathy (DON), or with DON as determined by the investigator at screening;
• Patients with corneal ulcers that are not relieved after treatment at the investigator's discretion;
• Scheduled orbital radiotherapy at any time before baseline or during the study, or surgical treatment for TED, including orbital decompression, strabismus surgery, and eyelid surgery;
• Participants with poorly controlled thyroid function, defined as free triiodothyronine (FT3) or free thyroxine (FT4) deviating from the normal reference range of the local laboratory by more than 50% at screening;
• Any other pre-existing disease, metabolic disorder, or physical examination or clinical laboratory abnormality that leads to a reasonable suspicion of a disease or condition that contraindicates the use of the investigational drug, affects the interpretation of study results, or places the participant at high risk of treatment complications;
• History of tinnitus or other hearing impairment in either ear during the screening period; or abnormal pure tone audiometry results (defined as an average bone conduction hearing threshold of ≥ 25 dB at 0.5, 1, 2, and 4 kHz, or a bone conduction hearing threshold of ≥ 40 dB at any frequency);
• Poorly controlled diabetes mellitus (defined as glycosylated hemoglobin ≥ 8.0% at screening);
• Cumulative dose of glucocorticoids used to treat TED ≥ 1 g methylprednisolone equivalents at any time before baseline;
• Oral or intravenous glucocorticoids within 30 days prior to screening;
• Peribulbar/periorbital injection of glucocorticoids within 90 days prior to screening;
• Oral or intravenous administration of any other non-steroidal immunosuppressants within 90 days prior to screening;
• Use of glucocorticoid eye drops/ointments or use of non-steroidal immunosuppressant eye drops within 30 days prior to screening;
• Received TEPEZZA or Teprotumumab N01 Injection at any time before screening;

Sponsors & Collaborators

• Innovent Biologics (Suzhou) Co. Ltd.: lead

Study Sites (1)

Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, 200125, China

Location

MeSH Terms

Conditions

Graves Ophthalmopathy

Interventions

Methylprednisolone

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

Intervention Hierarchy (Ancestors)

Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Central Study Contacts

Lily Zhang

CONTACT

13603216253; lily.zhang@innoventbio.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 4, 2025
• Study Start: December 30, 2025
• Primary Completion (Estimated): September 12, 2026
• Study Completion (Estimated): September 4, 2027
• Last Updated: December 4, 2025

Record last verified: 2025-11

Locations

CN (1)