Active Brain & Nervous System NIHR-supported project Digestion, Kidneys & Other Organs

A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Thyroid Eye Disease

Funder NIHR (part of DHSC)
Recipient organisation NIHR Guy's and St Thomas' Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Feb 2028

In plain English

AI plain-English summary

A drug already approved for a rare muscle-weakening disease is now being tested in a late-stage clinical trial to see if it can stop the immune system from attacking the tissue behind the eyes in thyroid eye disease. Thyroid eye disease causes the eyelids to retract and the eyes to bulge forward, sometimes severely enough to double vision or compress the optic nerve. Current treatments are limited: steroids can reduce inflammation but often cause side effects, and surgery is invasive. The problem is driven by rogue antibodies that bind to receptors on cells behind the eye. The drug, efgartigimod, is a modified fragment of a human antibody designed to latch onto a protein called FcRn, which normally recycles antibodies and keeps their levels high. By blocking FcRn, efgartigimod forces the body to break down harmful antibodies more quickly, including those driving thyroid eye disease. If this trial succeeds, efgartigimod could become the first targeted therapy for thyroid eye disease that directly lowers the causative antibodies rather than just dampening general inflammation. That would give patients a non-surgical option to prevent vision loss and correct disfigurement. The study is randomised, double-masked, and placebo-controlled, and will last one to two years per participant.

View original technical description
This study aims to look at the safety and effect (efficacy, or how well it works) of the study drug (Efgartigimod) in people with thyroid eye disease (TED). The study will research how the study drug is absorbed and removed from the body. It will also explore how the immune system reacts to the study drug. Efgartigimod is a piece of a human antibody modified to bind better to a specific protein called FcRn. Antibodies are proteins that fight and prevent infections. FcRn helps maintain antibody levels. Efgartigimod is similar to antibodies naturally present in the human body. It is called a “biological” drug because it is a piece of protein produced in the cells of mammals. Efgartigimod may help improve TED. This is because efgartigimod may reduce the antibodies that cause symptoms in patients with TED. Efgartigimod has been approved as a treatment for adult patients with myasthenia gravis, a rare disease causing muscle weakness. Efgartigimod is also being studied as a treatment for other autoimmune conditions (conditions in which your immune system attacks part of your body). It is considered investigational because health authorities have not approved it for the treatment of TED. The study will last about 1 to 2 years depending on how participants individually respond to the study drug.

Researchers

Susie Morley (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory DemyeliN/Ating Polyneuropathy (CIDP). (ADHERE)
ARGX-113-2308 - A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
A Phase 3, Multi-center, Randomized, Quadruple-masked, Placebo-controlled Study of Batoclimab for the Treatment of Participants with Active Thyroid Eye Disease (TED)
A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
IMVT-1401-3201 - A Phase 3, Multi-center, Randomized, Quadruple-masked, Placebo-controlled Study of Batoclimab for the Treatment of Participants with Active Thyroid Eye Disease (TED)

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