Clinical trial NCT06389578: A Study of TEPEZZA® Treatment in Participants With Thyroid Eye Disease
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Thyroid Eye Disease
Keywords: Thyroid Eye Disease
TEPEZZA
Teprotumumab
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2024-04-29
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Pharmacokinetics (PK): Area Under the Curve (AUC) of Teprotumumab
timeFrame: Pre dose through Week 6
measure: PK: Maximum Serum Concentration (Cmax) of Teprotumumab
timeFrame: Pre dose through Week 6
measure: Number of Participants With Adverse Events (AE)
timeFrame: Up to Week 6
Secondary outcomes:
Other outcomes:
Locations:
city: Miami
country: United States
facility: Bascom Palmer Eye Institute - University of Miami
state: Florida
city: Saint Louis
country: United States
facility: Barnes Jewish Hospital Washington University
state: Missouri
city: Bellaire
country: United States
facility: Neuro-Eye Clinical Trials
state: Texas
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: