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Thyroid Eye DiseasePhase 4NCT04583735

A Study Evaluating TEPEZZA® Treatment in Patients With Chronic (Inactive) Thyroid Eye Disease

A Phase 4 study testing TEPEZZA in people with Thyroid Eye Disease and Chronic (Inactive) Thyroid Eye Disease, run by Amgen at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The overall objective is to investigate the efficacy, safety and tolerability of TEPEZZA® in participants with chronic (inactive) TED (thyroid eye disease). Approximately 57 participants will be enrolled. There will be a treatment period (through Week 24) and a follow up period (where TEPEZZA will not be infused). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
TEPEZZA
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
62 participants
Where
12 sitesUnited States
Study dates
2 Sep 2021to 12 Oct 2023
Phase
Phase 4
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Written informed consent.
  • Male or female at least 18 years old at Screening.
  • Initial diagnosis of TED ≥2 years but <10 years prior to Screening. Clinical diagnosis of stable, chronic (inactive) TED, as determined by participant medical records indicating a Clinical Activity Score (CAS) ≤1 in both eyes for at least 1 year prior to Screening or all of the following:
  • no progression in proptosis for at least 1 year prior to Screening
  • if participant has history of diplopia due to TED, no progression in diplopia for at least 1 year prior to Screening
  • no new inflammatory TED symptoms for at least 1 year prior to Screening
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  • CAS ≤1 at the Screening and Baseline Visits.
  • Proptosis ≥3-mm increase from participant's Baseline (prior to diagnosis of TED), as estimated by treating physician and/or proptosis ≥3 mm above normal for race and gender.
  • Participants must be euthyroid with the participant's Baseline disease under control or have mild hypo- or hyperthyroidism (defined as free thyroxine and free triiodothyronine 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.
  • Does not require immediate surgical ophthalmological intervention and is not planning corrective surgery/irradiation during the course of the trial.
  • Diabetic participants must have HbA1c ≤8.0% at Screening.
  • Participants with a history of inflammatory bowel disease, ulcerative colitis or Crohn's disease must be in clinical remission for at least 3 months, with no history of bowel surgery within 6 months prior to screening and no planned surgery during the trial. Concomitant stable therapies for inflammatory bowel disease without modifications in the 3 months prior to Screening are allowed.
  • Women of childbearing potential (including those with an onset of menopause <2 years prior to Screening, non-therapy-induced amenorrhea for <12 months prior to Screening, or not surgically sterile [absence of ovaries and/or uterus]) must have a negative serum pregnancy test at Screening and negative urine pregnancy tests at all protocol-specified time points (i.e., prior to each dose and throughout participant's participation); participants who are sexually active with a non-vasectomized male partner must agree to use 2 reliable forms of contraception during the trial, 1 of which is recommended to be hormonal, such as an oral contraceptive. Hormonal contraception must be started at least 1 full cycle prior to Baseline and continue for 180 days after the last dose of trial drug. Highly effective contraceptive methods (failure rate <1% per year), when used consistently and correctly, include implants, injectables, combination oral contraceptives, some intrauterine devices, sexual abstinence and vasectomized partner.
  • Willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the trial.

Who cannot join

  • Decreased best-corrected visual acuity due to optic neuropathy, defined by a decrease in vision of 2 lines on the Snellen chart, new visual field defect or color defect secondary to optic nerve involvement 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.
  • Prior orbital irradiation, orbital decompression in the study eye.
  • Prior strabismus surgery.
  • Alanine aminotransferase or aspartate aminotransferase >3 × the upper limit of normal or estimated glomerular filtration rate ≤30 mL/min/1.73 m2 at Screening.
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  • 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 (Rituxan® or MabThera®) within 12 months prior to the first infusion of trial drug or tocilizumab (Actemra® or Roactemra®) within 6 months prior to the first infusion of trial drug. Use of any other non-steroid immunosuppressive agent within 3 months prior to the first infusion of trial drug.
  • Any previous treatment with TEPEZZA, including previous enrollment in this trial or participation in a prior teprotumumab trial.
  • Treatment with any mAb within 3 months prior to Screening.
  • Identified pre-existing ophthalmic disease that, in the judgment of the Investigator, would preclude trial participation or complicate interpretation of trial results.
  • Use of an investigational agent for any condition within 60 days or 5 half-lives, whichever is longer, prior to Screening or anticipated use during the course of the trial.
  • Malignant condition in the past 12 months (except successfully treated basal/squamous cell carcinoma of the skin or cervical cancer in situ).
  • Pregnant or lactating women.
  • Current drug or alcohol abuse or history of either within the previous 2 years, in the opinion of the Investigator or as reported by the participant.
  • Known hypersensitivity to any of the components of TEPEZZA or prior hypersensitivity reactions to mAbs.
  • Poorly controlled human immunodeficiency virus infection or untreated or positive viral load for hepatitis C or hepatitis B infections.
  • Any other condition that, in the opinion of the Investigator, would preclude inclusion in the trial.

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

United States12 sites

  • Univ of Colorado Dept of Ophthalmology

    Aurora, Colorado, 80045

  • The Private Practice of Raymond Douglas

    Beverly Hills, California, 90210

  • Endocrinology Specialists & Thyroid Center

    Greenville, South Carolina, 29605

  • Neuro-Eye Clinical Trials

    Houston, Texas, 77401

  • Perlman Medical Offices / UCSD

    La Jolla, California, 92037

  • Las Vegas Endocrinology

    Las Vegas, Nevada, 89074

  • MACRO Trials

    Los Angeles, California, 90048

  • Hamilton Eye Institute / U Tennessee

    Memphis, Tennessee, 38163

  • Bascom Palmer Eye Institute / Univ of Miami

    Miami, Florida, 33136

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 52336

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Washington University

    St Louis, Missouri, 63108

Other trials in United States

About this study

Official title: A Phase 4, Randomized, Double-masked, Placebo-controlled, Multicenter Trial to Evaluate the Efficacy and Safety of TEPEZZA® in Treating Patients With Chronic (Inactive) Thyroid Eye Disease

The overall objective is to investigate the efficacy, safety and tolerability of TEPEZZA® in participants with chronic (inactive) TED (thyroid eye disease). Approximately 57 participants will be enrolled. There will be a treatment period (through Week 24) and a follow up period (where TEPEZZA will not be infused).

Read the detailed description

This is a randomized, double-masked, placebo-controlled, parallel-group, multicenter trial. Participants will be screened for the trial within 4 weeks prior to Baseline (Day 1). Approximately 57 participants who meet the trial eligibility criteria will be randomized on Day 1 in a 2:1 ratio to receive 8 infusions of TEPEZZA or placebo once every 3 weeks.

All participants will enter a 24-week double-masked Treatment Period, during which trial drug will be infused on Day 1 (Baseline) and Weeks 3, 6, 9, 12, 15, 18 and 21 (with a final visit at Week 24 of the 24-week Treatment Period). At the end of the double-masked Treatment Period (Week 24), all patients will be assessed for treatment response. Non-responders may choose to receive 8 infusions of TEPEZZA in an open-label fashion q3W at Weeks 24, 27, 30, 33, 36, 39, 42 and 45.

Study acquired from Horizon in 2024.

Being tested
TEPEZZA, Placebo
Keywords
Proptosis
Medical subject headings
Graves Ophthalmopathy, Bronchiolitis Obliterans Syndrome, Sedentary Behavior, Exophthalmos

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TEPEZZABiological

    Also called teprotumumab-trbw, HZN-001

    Intravenous infusion

  • PlaceboDrug

    Also called sodium chloride

    Intravenous infusion

Study groups in NCT04583735
Study groupRoleWhat they receive
TEPEZZAOtherParticipants received intravenous infusion of 10 milligrams per kilogram (mg/kg) teprotumumab at first infusion and then 20 mg/kg once every 3 weeks (Q3W) for next 7 infusions during the double masked treatment period. Proptosis non-responders who completed the double-masked treatment period had opted to receive 10 mg/kg teprotumumab at first infusion and then 20 mg/kg Q3W for next 7 infusions during open label treatment period.
Receives: Biological: TEPEZZA
PlaceboPlacebo comparatorParticipants received teprotumumab matching placebo by intravenous infusion, Q3W for 8 infusions during the double masked treatment period. Proptosis non-responders who completed the double-masked treatment period had opted to receive 10 mg/kg teprotumumab at first infusion and then 20 mg/kg Q3W for next 7 infusions during open label treatment period.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Proptosis of Study Eye at Week 24

    Proptosis assessments were performed using a Hertel exophthalmometer. It measures the anterior projection of the eye from the lateral orbital rim to the cornea (proptosis).

    Measured: Baseline, week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Oct 2020
  2. Study start2 Sep 2021
  3. Primary completion17 Mar 2023
  4. Study completion12 Oct 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
62 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04583735
Sponsor's protocol ID
HZNP-TEP-403
Status last verified
Jun 2024
First submitted
6 Oct 2020
Last updated
1 Jul 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Publications

  • Ugradar S, Kumar A, Karon M, Wang Z, Subramanian PS, Wester ST. Post Hoc MRI Analysis of Orbital Soft Tissue Volumes Pre- and Post-Teprotumumab in Patients with Chronic, Low-Activity Thyroid Eye Disease. Ophthalmol Ther. 2026 Oct 6. doi: 10.1007/s40123-026-01516-9. Online ahead of print.

    PubMed 42837042

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing TEPEZZA in people with Thyroid Eye Disease and Chronic (Inactive) Thyroid Eye Disease, run by Amgen at 12 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 1 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Proptosis of Study Eye at Week 24 (time frame: Baseline, week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04583735, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2024.

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