Research and Clinical Trials for Thyroid Eye Disease

Medicine is based on research. Clinical trials using real people help decide the safety and efficacy of treatments. Research into thyroid eye disease (TED) is currently underway. Significant progress has been made in the last decade.

In 2020, the first and only drug for the treatment of TED was approved by the US Food and Drug Administration (FDA). This approval was made possible by clinical trials and their participants.¹

Thyroid eye disease and clinical trials

Because TED is rare, fewer people are affected by the condition. This means fewer people are available to participate in clinical trials. Whether or not you participate in a clinical trial is up to you. However, your doctor will likely ask you about your interest in participating in a trial because of TED’s rarity.

Stages of clinical trials

Before starting any clinical trial, experts must perform a lot of research. These experts make detailed and safe plans for their trials, known as protocols. There are 4 phases of clinical trials.²

Phase 1 uses a small group of people to test the safety and dosage of a drug. Most of the time, these participants are healthy volunteers without the targeted condition. The purpose of phase 1 testing is to see how the drug interacts in the body and what dosages are safe. Before starting phase 1 trials, researchers often use animal models to estimate starting doses.²

Phases 2 and 3 usually have the same goal. These phases involve people with the targeted condition. Both phases look to see how effective the drug is and monitor for side effects. Phase 3 studies often involve more people than phase 2 studies.²

Phase 4 studies occur after a drug has been approved. This allows researchers to monitor long-term effects.²

Emerging TED research

Research findings have helped researchers, doctors, and TED specialists to understand the underlying causes and effects of TED. With this information, researchers often look at how drugs used for other conditions might help treat TED symptoms.

An off-label drug is a drug that is used to treat a different condition than the one it was approved by the FDA to treat. This also means the FDA has not yet determined the drug is safe and effective when used the way you are taking it.³

Off-label does not mean the drug is unsafe to take. Often, it means there has not been an official clinical trial to test the drug for this specific rare disease. You should always talk to your doctor about any questions you have about your treatments.³

Off-label drugs that may be used to treat TED include:^4-9

• Cosentyx® (secukinumab)
• CellCept® (mycophenolate mofetil/MMF)
• Rituxan® (rituximab)
• Actemra® (tocilizumab)

One drug, IMVT-1401, had clinical studies halted in 2021. Clinical studies may continue at a later date to prove its efficacy and safety in those with TED.¹⁰

Finding TED clinical trials

Ask your doctor if you are interested in participating in a clinical trial. Your TED specialist is an especially good resource. These specialists know of trials both in your area and afar that you can participate in. You can also view current clinical trials both in the United States and around the world at ClinicalTrials.gov.

If you find a trial you are interested in, ask your TED specialist.