A Study of TEPEZZA® Treatment in Participants With Thyroid Eye Disease

NCT06389578 | Completed Phase 1 FDA Drug

• 1 other identifier: HZNP-TEP-103
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 3

Brief Summary

The main goal of this study is to learn how teprotumumab will be processed in the body (Pharmacokinetics) subcutaneously and whether it is safe and tolerable after administration into adult patients with thyroid eye disease.

Conditions

Thyroid Eye Disease

Keywords

Thyroid Eye Disease; TEPEZZA; Teprotumumab

Outcome Measures

Primary Outcomes (3)

• Pharmacokinetics (PK): Area Under the Curve (AUC) of Teprotumumab

  AUC will be evaluated from the collected PK samples.

  Pre dose through Week 6

• PK: Maximum Serum Concentration (Cmax) of Teprotumumab

  Cmax will be evaluated from the collected PK samples.

  Pre dose through Week 6

• Number of Participants With Adverse Events (AE)

  An AE is any untoward medical occurrence in a participant or clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product.

  Up to Week 6

Study Arms (2)

Cohort 1

EXPERIMENTAL

Participants will receive a single dose of lyophilized teprotumumab by subcutaneous (SC) administration on Day 1 followed by intravenous (IV) infusions of teprotumumab at the approved dosing regimen.

Drug: Teprotumumab

Cohort 2

EXPERIMENTAL

Participants will receive a single high concentration formulation teprotumumab by SC administration on Day 1 followed by IV infusions of teprotumumab at the approved dosing regimen.

Drug: Teprotumumab

Interventions

Teprotumumab DRUG

SC administration and IV infusion

Also known as: Tepezza

Cohort 1; Cohort 2

Eligibility Criteria

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

You may qualify if:

• Thyroid eye disease (not sight-threatening but has an appreciable impact on daily life), usually associated with one or more of the following: lid retraction ≥ 2 mm, moderate or severe soft tissue involvement, proptosis, and/or inconstant or constant diplopia.
• Proptosis ≥ 3 mm increase from the participant's baseline (prior to diagnosis of TED), as estimated by treating physician and/or proptosis ≥ 3 mm above normal for race and gender.
• Participant must be euthyroid with baseline disease under control or have mild hypo- or hyperthyroidism 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.
• Does not require immediate surgical ophthalmological intervention.
• Participants with diabetes must have HbA1c ≤ 8.0% at Screening.

You may not qualify if:

• Decreased best-corrected visual acuity due to optic neuropathy within the last 6 months.
• Corneal decompensation unresponsive to medical management in the study eye.
• Decrease in proptosis of ≥ 2 mm in the study eye between Screening and Baseline.
• Alanine aminotransferase or aspartate aminotransferase \> 3x the upper limit of normal or estimated glomerular filtration rate ≤ 30 mL/min/1.73 m2 at Screening.
• Use of any steroid (IV, oral, steroid eye drops) for the treatment of TED or other conditions within 3 weeks prior to Screening. Steroids cannot be initiated during the trial. Exceptions include topical and inhaled steroids and steroids used to treat infusion reactions.
• Any treatment with rituximab, tocilizumab, or any other non-steroid immunosuppressive agent within 90 days prior to the first injection of investigational product on Day 1.
• Any previous treatment with HZN-001 or TEPEZZA (teprotumumab-trbw), including previous enrollment in this trial or participation in a prior TEPEZZA trial.
• Treatment with any mAb within 3 months prior to Screening.

Sponsors & Collaborators

• Amgen: lead

Study Sites (3)

Bascom Palmer Eye Institute - University of Miami

Miami, Florida, 33136, United States

Location

Barnes Jewish Hospital Washington University

St Louis, Missouri, 63108, United States

Location

Neuro-Eye Clinical Trials

Bellaire, Texas, 77401, United States

Location

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 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 24, 2024
• First Posted: April 29, 2024
• Study Start: July 14, 2022
• Primary Completion: February 28, 2023
• Study Completion: September 12, 2023
• Last Updated: June 21, 2024

Record last verified: 2024-06

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

US (3)