Teprotumumab N01 Versus Methylprednisolone After Urgent Orbital Decompression for Dysthyroid Optic Neuropathy

NCT07594912 | Not Yet Recruiting Phase 2

• 1 other identifier: yanghs20250429
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to evaluate the efficacy and safety of sequential Teprotumumab N01 compared with intravenous methylprednisolone (IVMP) after urgent orbital decompression in patients with dysthyroid optic neuropathy (DON).

Conditions

Thyroid Eye Disease, TED; Graves Ophthalmopathy; Optic Neuropathy

Keywords

Dysthyroid optic neuropathy; Orbital decompression; Teprotumumab N01; Intravenous methylprednisolone pulse; Thyroid eye disease

Outcome Measures

Primary Outcomes (1)

• Change in Best-Corrected Visual Acuity (BCVA) From Baseline to Week 24

  BCVA will be assessed using a Snellen visual acuity chart and converted to logarithm of the minimum angle of resolution (logMAR) units for analysis. Lower logMAR values indicate better visual acuity. A negative change from baseline indicates improvement.

  Baseline to Week 24

Secondary Outcomes (8)

• Change in BCVA From Baseline to Week 12

  Baseline to Week 12

• Change in Visual Field Mean Deviation (VF-MD) Measured by Humphrey Field Analyzer

  Baseline to Weeks 12 and 24

• Clinical Activity Score (CAS)

  Baseline to Weeks 12 and 24

• Proptosis Measured by Hertel Exophthalmometry

  Baseline to Weeks 12 and 24

• Change in Gorman Diplopia Score

  Baseline to Weeks 12 and 24

Study Arms (2)

Postoperative Sequential Teprotumumab N01

EXPERIMENTAL

Participants in this arm will undergo urgent orbital decompression and then receive postoperative sequential Teprotumumab N01.

Drug: Teprotumumab N01

Postoperative Sequential Intravenous Methylprednisolone

ACTIVE COMPARATOR

Participants in this arm will undergo urgent orbital decompression and then receive postoperative sequential intravenous methylprednisolone.

Drug: Intravenous Methylprednisolone (IVMP)

Interventions

Teprotumumab N01 DRUG

Teprotumumab N01 will be administered intravenously after urgent orbital decompression. The first infusion will be 10 mg/kg, followed by 20 mg/kg every 3 weeks for 7 additional infusions.

Also known as: Teprotumumab, Insulin-like growth factor-1 receptor monoclonal antibody, IGF-1R inhibitor

Postoperative Sequential Teprotumumab N01

Intravenous Methylprednisolone (IVMP) DRUG

IVMP will be administered after urgent orbital decompression at 0.5 to 1.0 g per day for 3 consecutive days, followed by oral prednisone tapering according to the participant's clinical status and investigator judgment.

Also known as: Methylprednisolone, Methylprednisolone sodium succinate, Intravenous methylprednisolone pulse therapy, IVMP

Postoperative Sequential Intravenous Methylprednisolone

Eligibility Criteria

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

You may qualify if:

• Able and willing to provide written informed consent.
• Age 18 to 65 years.
• Clinical diagnosis of thyroid eye disease (TED) with dysthyroid optic neuropathy (DON). Diagnosis of DON must meet both of the following criteria:
• Visual dysfunction not explained by other causes, such as decreased best-corrected visual acuity, visual field defect, abnormal color vision, or relative afferent pupillary defect.
• Imaging evidence of orbital apex crowding, optic nerve compression, or optic nerve stretching.
• The study eye meets the criteria for urgent orbital decompression, defined as either of the following:
• Poor response after rescue intravenous methylprednisolone for the current dysthyroid optic neuropathy episode, with a cumulative methylprednisolone-equivalent dose of no more than 3.0 g, defined as no improvement or continued deterioration of visual function within 1 to 2 weeks.
• Contraindication to glucocorticoids or investigator judgment that direct urgent mechanical decompression is required.
• Thyroid function is normal or mildly abnormal before enrollment, with free triiodothyronine (FT3) and free thyroxine (FT4) within 50% above or below the normal reference range when possible.
• Alanine aminotransferase (ALT) no more than 1.5 times the upper limit of normal, aspartate aminotransferase (AST) no more than 3 times the upper limit of normal, and serum creatinine no more than 1.5 times the upper limit of normal.
• For participants with diabetes mellitus, hemoglobin A1c (HbA1c) less than 9.0% and stable antidiabetic treatment within 60 days before enrollment.
• For women of childbearing potential, a pregnancy test must be negative before enrollment.
• Participants of reproductive potential agree to use effective contraception during the study and for 90 days after the last dose of study treatment.
• Able and willing to comply with study treatment and follow-up procedures.

You may not qualify if:

• Orbital decompression surgery within 6 months before screening.
• Bilateral dysthyroid optic neuropathy requiring urgent bilateral orbital decompression at baseline.
• Cumulative preoperative methylprednisolone-equivalent dose greater than 3.0 g for the current dysthyroid optic neuropathy episode.
• Systemic glucocorticoid treatment for non-thyroid eye disease within 3 months before screening with cumulative methylprednisolone-equivalent dose of 1.0 g or greater.
• Tocilizumab or other systemic immunosuppressive treatment within 3 months before screening, or rituximab within 6 months before screening.
• Orbital radiotherapy within 3 months before screening.
• Previous treatment with teprotumumab.
• Other ocular diseases that may significantly affect visual function assessment, including glaucomatous optic neuropathy, macular disease, severe cataract, or non-thyroid eye disease optic neuropathy.
• Irreversible optic nerve damage in the study eye with very low potential for visual recovery, as judged by the investigator.
• History of definite inner ear disease or clinically significant hearing impairment.
• Severe cardiovascular disease.
• Severe hepatic or renal disease.
• Active infection or clinically significant infectious disease, including active hepatitis, HIV infection, syphilis, or active tuberculosis.
• Active gastrointestinal ulcer.
• Clinically significant abnormal blood test results, including white blood cell count less than 4.0 × 10\^9/L, platelet count less than 80 × 10\^9/L, hemoglobin less than 110 g/L in males or less than 100 g/L in females.

Sponsors & Collaborators

• Sun Yat-sen University: lead

Study Sites (1)

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, 510000, China

Location

MeSH Terms

Conditions

Graves Ophthalmopathy; Optic Nerve Diseases

Interventions

teprotumumab; Methylprednisolone; Methylprednisolone Hemisuccinate

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; Cranial Nerve Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

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

Central Study Contacts

Hui jing Ye, M.D, PhD

CONTACT

+8620-87331539; yehuijing@qq.com

Gesang Zhuoma

CONTACT

+86-18008941211; 1139148653@qq.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: May 5, 2026
• First Posted: May 19, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): April 1, 2028
• Last Updated: May 19, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)