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

Phase 1
Completed
Conditions
Thyroid Eye Disease
Interventions
Registration Number
NCT06389578
Lead Sponsor
Amgen
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.

Detailed Description

This is a Phase 1b, open-label, multicenter trial to assess the PK, safety, and tolerability of a single SC dose of lyophilized teprotumumab and high concentration formulation teprotumumab in participants with thyroid eye disease. Approximately 6 participants (in Cohort 1) and approximately 10 participants (in Cohort 2) who meet the trial eligibility criteria will be enrolled in a 1:1 ratio.

Study acquired from Horizon in 2024.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
16
Inclusion Criteria
  1. 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.
  2. 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.
  3. 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.
  4. Does not require immediate surgical ophthalmological intervention.
  5. Participants with diabetes must have HbA1c ≤ 8.0% at Screening.
Exclusion Criteria
  1. Decreased best-corrected visual acuity due to optic neuropathy within the last 6 months.
  2. Corneal decompensation unresponsive to medical management in the study eye.
  3. Decrease in proptosis of ≥ 2 mm in the study eye between Screening and Baseline.
  4. Alanine aminotransferase or aspartate aminotransferase > 3x the upper limit of normal or estimated glomerular filtration rate ≤ 30 mL/min/1.73 m2 at Screening.
  5. 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.
  6. 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.
  7. Any previous treatment with HZN-001 or TEPEZZA (teprotumumab-trbw), including previous enrollment in this trial or participation in a prior TEPEZZA trial.
  8. Treatment with any mAb within 3 months prior to Screening.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Cohort 2TeprotumumabParticipants 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.
Cohort 1TeprotumumabParticipants 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.
Primary Outcome Measures
NameTimeMethod
Pharmacokinetics (PK): Area Under the Curve (AUC) of TeprotumumabPre dose through Week 6

AUC will be evaluated from the collected PK samples.

PK: Maximum Serum Concentration (Cmax) of TeprotumumabPre dose through Week 6

Cmax will be evaluated from the collected PK samples.

Number of Participants With Adverse Events (AE)Up to Week 6

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.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (3)

Bascom Palmer Eye Institute - University of Miami

🇺🇸

Miami, Florida, United States

Barnes Jewish Hospital Washington University

🇺🇸

Saint Louis, Missouri, United States

Neuro-Eye Clinical Trials

🇺🇸

Bellaire, Texas, United States

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