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

Thyroid Eye Disease Clinical Trial

Official title:

A Phase 1b, Open-label, Multicenter Trial to Evaluate the Pharmacokinetics, Safety, and Tolerability of Two Formulations of a Single Subcutaneous Dose of Teprotumumab Followed by Multiple Intravenous Doses of TEPEZZA in Participants With Thyroid Eye Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06389578
• Other study ID #: HZNP-TEP-103
• Status: Completed
• Phase: Phase 1
• Start date: July 14, 2022
• Est. completion date: September 12, 2023

Study information

• Verified date: April 2024
• Source: Horizon Pharma USA, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: September 12, 2023
• Est. primary completion date: February 28, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Eye Diseases
• Graves Ophthalmopathy
• Thyroid Diseases
• Thyroid Eye Disease

Intervention

Drug:
• Teprotumumab
SC administration and IV infusion

Locations

Country	Name	City	State
United States	Neuro-Eye Clinical Trials	Bellaire	Texas
United States	Bascom Palmer Eye Institute - University of Miami	Miami	Florida
United States	Barnes Jewish Hospital Washington University	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Horizon Therapeutics USA, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pharmacokinetics (PK): Area Under the Curve (AUC) of Teprotumumab	AUC will be evaluated from the collected PK samples.	Pre dose through Week 6
Primary	PK: Maximum Serum Concentration (Cmax) of Teprotumumab	Cmax will be evaluated from the collected PK samples.	Pre dose through Week 6
Primary	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