Teprotumumab for Thyroid Eye Disease

Not currently recruiting at 50 trial locations

Overseen By Horizon Therapeutics

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Amgen

Disqualifiers: Immediate surgery, Diabetes, IBD, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests teprotumumab, a medication for Thyroid Eye Disease (TED), to evaluate its safety, effectiveness, and the necessity of different treatment durations. The trial includes three groups, each receiving varying numbers of teprotumumab infusions. Researchers will analyze blood samples for disease biomarkers. Suitable candidates for this trial are adults diagnosed with TED within the last seven years, who exhibit noticeable eye bulging (proptosis) and have stable thyroid levels. As a Phase 4 trial, teprotumumab has already received FDA approval and proven effective; this research aims to understand how it benefits more patients.

Do I have to stop taking my current medications for the trial?

The trial requires that you stop taking steroids for TED or other conditions at least 3 weeks before screening and during the trial, except for topical and inhaled steroids or those used for infusion reactions. Other immunosuppressive agents must be stopped 3 months before the first infusion of teprotumumab.

What is the safety track record for teprotumumab?

Research has shown that teprotumumab, a treatment for Thyroid Eye Disease, is usually well-tolerated, though some side effects can occur. Common side effects include muscle cramps, nausea, hair loss, diarrhea, fatigue, and high blood sugar. Some individuals also experience hearing issues and changes in taste. These side effects occur in at least 5% of participants and are more frequent than with a placebo. Understanding these potential reactions and discussing them with a doctor is important when considering participation in a clinical trial. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Teprotumumab stands out for treating Thyroid Eye Disease because it specifically targets and inhibits the insulin-like growth factor-1 receptor (IGF-1R), a unique mechanism compared to traditional treatments like corticosteroids or surgical options. This targeted approach can directly reduce the inflammation and swelling behind the eyes, potentially offering a more effective and less invasive alternative. Researchers are excited because teprotumumab has shown promise in rapidly improving symptoms, providing relief in situations where other options might take longer to work or involve more significant side effects.

What is the effectiveness track record for teprotumumab in treating Thyroid Eye Disease?

Research has shown that teprotumumab effectively treats Thyroid Eye Disease (TED). Studies have found that it significantly reduces eye bulging and lowers the Clinical Activity Score, which measures disease activity. Nearly 70% of patients experienced improvement in double vision. Inflammatory symptoms and overall eye health improved in 90% of patients. In this trial, participants will receive different regimens of teprotumumab, with some receiving 4, 8, or 16 infusions, to evaluate its effectiveness further. These findings suggest that teprotumumab can provide significant relief for those suffering from TED. ^{6 7 8 9 10}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

Adults aged 18-80 with Thyroid Eye Disease (TED) diagnosed within the last 7 years, not needing immediate eye surgery. Women must test negative for pregnancy and use reliable contraception. Diabetics with HbA1c ≤8% are eligible if they have controlled thyroid levels or mild thyroid dysfunction.

Inclusion Criteria

My eye bulging has increased by 3 mm or more since my TED diagnosis.

I was diagnosed with TED within the last 7 years.

My thyroid function is normal or slightly off but under control.

Exclusion Criteria

You are allergic to teprotumumab or have had allergic reactions to similar medications in the past.

You have untreated HIV, or positive viral load for hepatitis C or hepatitis B infections.

I have had radiation, surgery to relieve eye pressure, or eye muscle surgery.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Initial Treatment

Participants receive teprotumumab infusions based on cohort assignment: 4, 8, or 16 infusions

24-48 weeks

Infusions every 3 weeks

End-of-Initial Treatment

Comprehensive assessment of treatment response at the end of the initial treatment period

1 week

1 visit (in-person)

Initial Follow-up

Participants are monitored for safety and effectiveness; proptosis responders and non-responders are assessed for re-treatment eligibility

52 weeks

Re-treatment (if applicable)

Eligible participants receive a second course of teprotumumab infusions if they flare during follow-up

24 weeks

Infusions every 3 weeks

Long-term Follow-up

Participants are monitored for long-term safety and effectiveness

Up to Week 136

What Are the Treatments Tested in This Trial?

Interventions

• Teprotumumab

Trial Overview The trial tests Teprotumumab's safety and effectiveness in treating TED compared to a placebo. It involves three different treatment durations, random assignment of participants to groups, double-masking, and parallel assignment across multiple centers.

How Is the Trial Designed?

Group I: Teprotumumab 8 Infusions Experimental Treatment 1 Intervention

8 infusions of teprotumumab (10 mg/kg for the first infusion and 20 mg/kg for the remaining 7 infusions) (Cohort 2)

Group II: Teprotumumab 4 Infusions Experimental Treatment 2 Interventions

• 4 infusions of teprotumumab (10 mg/kg for the first infusion and 20 mg/kg for the remaining 3 infusions) (Cohort 1) followed by 4 infusions of: * Placebo if a participant is a treatment responder at Week 12 or * Teprotumumab 20 mg/kg if a participant is a treatment non-responder at Week 12

Group III: Teprotumumab 16 Infusions Experimental Treatment 1 Intervention

16 infusions of teprotumumab (10 mg/kg for the first infusion and 20 mg/kg for the remaining 15 infusions) (Cohort 3)

Teprotumumab is already approved in United States for the following indications:

Approved in United States as Tepezza for:

• Thyroid Eye Disease

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Horizon Therapeutics USA, Inc.

Lead Sponsor

Trials

4

Recruited

420+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9312457/

Teprotumumab (Tepezza) for Thyroid Eye Disease - PMC The primary outcome measurement was a decrease in clinical activity score (CAS) of two or more points and proptosis reduction of 2mm or more. At 24 weeks, 29 ...

2. tepezzahcp.com tepezzahcp.com/about-tepezza/tepezza-efficacy

TEPEZZA® for HCPs - TEPEZZA® (teprotumumab-trbw) Efficacy In the Phase 4 trial in patients with chronic TED, no differences between the teprotumumab and placebo groups were observed for diplopia endpoints. The trial ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38824618/

Long-Term Efficacy of Teprotumumab in Thyroid Eye Disease Inflammatory and ophthalmic composite outcome improvements were seen in 90% of patients with nearly 70% reporting improvement in diplopia and ...

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa1910434

Teprotumumab for the Treatment of Active Thyroid Eye ... Teprotumumab resulted in better outcomes with respect to proptosis, Clinical Activity Score, diplopia, and quality of life than placebo; serious adverse events ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S1530891X24006542

Effects of Teprotumumab and Role of Human Leukocyte ... This study showed that teprotumumab significantly reduced thyroid stimulating immunoglobin, glycated hemoglobin A1c levels, proptosis, clinical activity score.

6. tepezzahcp.com tepezzahcp.com/about-tepezza/safety-and-adverse-reactions

Safety & Adverse Reactions - TEPEZZA (teprotumumab-trbw) The most common adverse reactions (incidence ≥5% and greater than placebo) are muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing ...

7. tepezza.com tepezza.com/about-tepezza/side-effects-and-safety

TEPEZZA Side Effects and Safety Information The most common side effects of TEPEZZA include muscle cramps or spasms, nausea, hair loss, diarrhea, feeling tired, high blood sugar, hearing problems, taste ...

8. tepezza.com tepezza.com/thyroid-eye-disease-support/side-effects-and-safety

TEPEZZA® (teprotumumab-trbw) Side Effects and Safety TEPEZZA is a prescription medicine used to treat Thyroid Eye Disease (TED), no matter if you've had TED for months or years.

9. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37925673/

Efficacy and Safety of Teprotumumab in Patients With ... Teprotumumab significantly improved proptosis vs placebo in longstanding/low inflammation TED, demonstrating efficacy regardless of disease duration/activity.

10. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10960718/

Teprotumumab-Related Adverse Events in Thyroid Eye ... The FDA has reported muscle spasms, hearing loss, hyperglycemia, nausea, alopecia, diarrhea, dry skin, dysgeusia, headache, and fatigue as systemic AEs ...