A Trial to Investigate Teprotumumab Subcutaneous Administration Compared With Placebo in Male and Female Adult Participants With Moderate-to-severe Active Thyroid Eye Disease

NCT06248619 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: HZNP-TEP-305
• Study Type: interventional
• Target: 89
• Locations: 11 countries
• Sites: 38

Brief Summary

The study consists of a randomized double-masked, placebo-controlled, parallel-group, multicenter trial with an optional open-label treatment period for proptosis non-responders who complete the Double-masked Treatment Period.

Conditions

Thyroid Eye Disease

Keywords

Teprotumumab; Active Thyroid Eye Disease; Tepezza; Teprotumumab-trbw

Outcome Measures

Primary Outcomes (1)

• Proptosis responder rate (percentage of participants with a ≥ 2-mm reduction from baseline in proptosis in the study eye without deterioration [≥ 2-mm increase] of proptosis in the fellow eye).

  Week 24

Secondary Outcomes (7)

• Mean change from Baseline in proptosis measurement in the study eye

  Week 24

• Overall responder rate (percentage of participants with ≥ 2-point reduction in CAS AND ≥ 2-mm reduction in proptosis from Baseline, provided there is no corresponding deterioration [≥ 2-point/mm increase] in CAS or proptosis in the fellow eye)

  Week 24

• Percentage of participants with a CAS value of 0 or 1

  Week 24

• Change from Baseline in diplopia as ordinal response categories

  Week 24

• Diplopia responder rate, defined as the percentage of participants with Baseline binocular diplopia > 0 who have a reduction of ≥ 1 grade

  Week 24

Study Arms (2)

Teprotumumab

EXPERIMENTAL

Teprotumumab administered SC

Biological: Teprotumumab

Placebo

PLACEBO COMPARATOR

Placebo for teprotumumab administered SC

Other: Placebo

Interventions

Teprotumumab BIOLOGICAL

SC injection

Teprotumumab

Placebo OTHER

SC injection

Placebo

Eligibility Criteria

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

You may qualify if:

• Participant must provide written informed consent.
• Participant can be male or female and must be between the ages of 18 and 80 years, inclusive, at Screening.
• Participant must have a clinical diagnosis of Graves' disease associated with active TED with a CAS ≥ 3 (on the 7-item scale) for the most severely affected eye at Screening and Baseline.
• Participant must have moderate-to-severe active TED (not sight-threatening but has an appreciable impact on daily life), usually associated with 1 or more of the following: lid retraction ≥ 2 mm, moderate or severe soft tissue involvement and/or inconstant or constant diplopia. (Note: Participants with no diplopia at Baseline will be limited to approximately 25% of the total number enrolled.)
• Participant has proptosis ≥ 3 mm from Baseline (prior to diagnosis of TED), as estimated by treating physician, and/or proptosis ≥ 3 mm above normal for race and gender.
• Participant had onset of active TED symptoms (as determined by participant records) within 15 months prior to Baseline.
• Participants must be euthyroid with the baseline disease under control or have mild hypoor hyperthyroidism (defined as free thyroxine \[FT4\] and free triiodothyronine \[FT3\] levels \< 50% above or below the normal limits) at Screening. Every effort should be made to correct the mild hypo- or hyperthyroidism promptly and to maintain the euthyroid state for the full duration of the trial.
• Participant does not require immediate surgical ophthalmological intervention and is not planning corrective surgery/irradiation during the trial.
• Women of childbearing potential must have a negative serum pregnancy test at Screening and negative urine pregnancy tests at all protocol-specified time points (ie, prior to each dose and throughout participation in the trial).
• Participant is willing and able to comply with the protocol requirements for the duration of the trial.

You may not qualify if:

• Participant has decreased best-corrected visual acuity due to optic neuropathy, defined by a decrease in vision of 2 lines on the Snellen chart (or equivalent), new visual field defect or color defect secondary to optic nerve involvement within the last 6 months.
• Participant has corneal decompensation unresponsive to medical management.
• Participant has a decrease in CAS of ≥ 2 points between Screening and Baseline.
• Participant has a decrease in proptosis of ≥ 2 mm between Screening and Baseline.
• Participant had prior orbital irradiation, orbital decompression or strabismus surgery (excluding childhood strabismus surgeries unrelated to TED/Graves' disease).
• Participant is planning to have eyelid surgery during the trial.
• Participant received periocular botulinum toxin injection within 12 months prior to Screening.
• Participant has any systemic use of a steroid (IV or oral) or steroid eye drops for the treatment of TED or other conditions within 3 weeks prior to Screening. Exceptions include local administration (excluding periocular), eg, topical, intra-articular, and inhaled steroids, as well as steroids used to treat infusion reactions.

Sponsors & Collaborators

• Amgen: lead

Study Sites (38)

Catalina Eye Care - NVISION - PPDS

Tucson, Arizona, 85712, United States

Location

Advanced Quality Medical Research

Orland Park, Illinois, 60462, United States

Location

W Kellogg Eye Center

Ann Arbor, Michigan, 48105-1912, United States

Location

Las Vegas Endocrinology

Henderson, Nevada, 89074, United States

Location

The Center for Eye and Facial Plastic Surgery

Somerset, New Jersey, 08773, United States

Location

Casey Eye Institute -515 SW Campus Dr

Portland, Oregon, 97239-3130, United States

Location

Scheie Eye Institute

Philadelphia, Pennsylvania, 19104-2640, United States

Location

University of Tennessee Health Science Center - 848 Adams Ave

Memphis, Tennessee, 38103-3452, United States

Location

Baylor College of Medicine-1977 Butler Blvd

Houston, Texas, 77030-4101, United States

Location

University of Washington Eye Institute

Seattle, Washington, 98104-2433, United States

Location

West Virginia University Eye Institute

Morgantown, West Virginia, 26506-1200, United States

Location

Hospital Universitario Austral

Pilar, Buenos Aires, B1629ODT, Argentina

Location

Organización Médica de Investigación

Buenos Aires, Ciudad Autónoma de BuenosAires, C1015ABO, Argentina

Location

Centro Medico Grupo Laser Vision

Rosario, Santa Fe Province, S2000, Argentina

Location

Sydney Eye Hospital

Sydney, New South Wales, 2000, Australia

Location

Queensland Eye Institute

Wooloongabba, Queensland, 4102, Australia

Location

Royal Adelaide Hospital

Adelaie, South Australia, 5000, Australia

Location

Centre For Eye Research Australia Ltd

East Melbourne, Victoria, 3002, Australia

Location

Vancouver Coastal Health Research Institute (VCHRI) - 2550 Willow St

Vancouver, British Columbia, V5Z 3N9, Canada

Location

McGill University Health Centre Research Institute

Montreal, Quebec, H4A 3J1, Canada

Location

CHU de Quebec-Universite Laval CUO Recherche Clinique Hopital du St-Sacrement

Québec, G1S 4L8, Canada

Location

AP-HM-Hôpital de La Conception

Marseille, Bouches-du-Rhône, 13010, France

Location

Universitätsklinikum Essen

Essen, North Rhine-Westphalia, 45147, Germany

Location

Azienda Ospedaliera Universitaria Federico II

Naples, Campania, 80131, Italy

Location

Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello

Pisa, 56124, Italy

Location

Azienda Ospedaliero Universitaria Pisana - Stabilimento Santa Chiara

Pisa, 56124, Italy

Location

Hayashi Eye Hospital

Fukuoka, Hukuoka, 812-0011, Japan

Location

Kozawa Eye hospital and Diabetes Center

Mito, Ibaraki, 310-0845, Japan

Location

Ishikawa Prefectural Central Hospital

Kanazawa, Ishikawa-ken, 920-8530, Japan

Location

University of Miyazaki Hospital

Miyazaki, Miyazaki, 889-1601, Japan

Location

Gokeikai Osaka Kaisei Hospital

Osaka-Shi Yodogawa-Ku, Ôsaka, 532-0003, Japan

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia-Avda de Fernando Abril Martorell 106

Valencia, 46026, Spain

Location

Changhua Christian Hospital

Changhua County, 50006, Taiwan

Location

Chung Shan Medical University Hospital

Taichung, Taiwan

Location

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

Moorfields Eye Hospital - PPDS

London, Middlesex, EC1V 2PD, United Kingdom

Location

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Graves Ophthalmopathy

Interventions

teprotumumab

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

• MD

  Amgen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2024
• First Posted: February 8, 2024
• Study Start: July 5, 2024
• Primary Completion: January 14, 2026
• Study Completion (Estimated): September 2, 2026
• Last Updated: January 28, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

IT (3); JP (5); AU (4); ES (3); TW (3); US (11); GB (1); FR (1); AR (3); DE (1); CA (3)