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

NCT ID: NCT07265258

Last Updated: 2025-12-04

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 92 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-30
• Study Completion Date: 2027-09-04

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Intravenous glucocorticoid (IVGC) group

Group Type: ACTIVE_COMPARATOR

Methylprednisolone

Intervention Type: 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.

Teprotumumab N01 group

Group Type: EXPERIMENTAL

Teprotumumab N01

Intervention Type: 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).

Interventions

Teprotumumab N01

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).

Intervention Type: BIOLOGICAL

Methylprednisolone

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Written informed consent.

2. Male or female subject between the ages of 18 and 80 years at screening.

3. Weight between 45 kg and 100 kg.

4. 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;

5. Exophthalmos ≥ 16 mm in the study eye at baseline.

6. 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.

Exclusion Criteria

Participants to be excluded (Participants meeting any of the following criteria will be regarded as ineligible):

1. 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;

2. Participants previously diagnosed with dysthyroid optic neuropathy (DON), or with DON as determined by the investigator at screening;

3. Patients with corneal ulcers that are not relieved after treatment at the investigator's discretion;

4. 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;

5. 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;

6. 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;

7. 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);

8. Poorly controlled diabetes mellitus (defined as glycosylated hemoglobin ≥ 8.0% at screening);

9. Cumulative dose of glucocorticoids used to treat TED ≥ 1 g methylprednisolone equivalents at any time before baseline;

10. Oral or intravenous glucocorticoids within 30 days prior to screening;

11. Peribulbar/periorbital injection of glucocorticoids within 90 days prior to screening;

12. Oral or intravenous administration of any other non-steroidal immunosuppressants within 90 days prior to screening;

13. Use of glucocorticoid eye drops/ointments or use of non-steroidal immunosuppressant eye drops within 30 days prior to screening;

14. Received TEPEZZA or Teprotumumab N01 Injection at any time before screening;

15. Received CD20 antibody or interleukin-6 receptor (IL-6R) antibody at any time before screening;

16. Have received any other TED therapeutic drugs under development (including but not limited to biologics targeting IGF-1R, FcRn, and TSHR) at any time before screening;

17. Use of any other monoclonal antibody within 90 days prior to screening;

18. Female participants in pregnancy or lactation.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Innovent Biologics (Suzhou) Co. Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Shanghai, Shanghai Municipality, China

Countries

China

Central Contacts

Lily Zhang

Role: CONTACT

lily.zhang@innoventbio.com

13603216253

Facility Contacts

Xianqun Fan

Role: primary

fanxq@sh163.net

Huifang Zhou

Role: backup

fangzzfang@sjtu.edu.cn

+86 13661901886

Other Identifiers

CIBI311A303

Identifier Type: -

Identifier Source: org_study_id