Azienda Ospedaliero-Universitaria Sant Andre
Rome, Italy
Rare diseases
Investigational molecules
Locations
A plain-language summary of the goals, design and what participants do
This study is being conducted to evaluate the safety and effectiveness of different treatment approaches for Myasthenia Gravis. This is a rare autoimmune disease where the body's immune system mistakenly attacks the connection between nerves and muscles, leading to muscle weakness. Specifically, this research focuses on a subtype called AChR-Ab Seropositive Generalized Myasthenia Gravis, which means the disease is present throughout the body and certain antibodies, known as acetylcholine receptor antibodies, can be detected in the blood.
The research involves the use of two medications: efgartigimod alfa and empasiprubart. Both of these are administered through an IV infusion, which is a method of delivering medication directly into a vein. Some participants will receive efgartigimod alfa alone, while others will receive empasiprubart as an additional treatment added to their current efgartigimod alfa therapy. This approach is intended for individuals who have experienced only a partial improvement in their symptoms while using the first medication.
During the study, participants will follow a specific treatment schedule to observe how the medications affect their condition and overall health. Monitoring will include checking vital signs and conducting an ECG, which is a test that records the electrical activity of the heart, to ensure the treatments are tolerated well. The study aims to observe changes in how much the disease impacts daily activities and the severity of muscle weakness over time.
The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.
7 criteria
6 criteria
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Rome, Italy
Leuven, Belgium
Leiden, The Netherlands
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is an experimental medicine given through a vein (IV infusion) that is being studied to see if it can help improve symptoms in people with myasthenia gravis, specifically when used alongside another treatment.
is a treatment given through a vein (IV infusion) used to help manage myasthenia gravis by targeting specific parts of the immune system.
This medication is a concentrated liquid that is administered through a slow drip into a vein. Currently being studied in clinical trials, it is classified as a monoclonal antibody designed to treat autoimmune conditions like myasthenia gravis. It works by specifically targeting and attaching to certain harmful proteins in the immune system to prevent them from causing damage.
This medicine is provided as a liquid solution that is delivered directly into the bloodstream through a vein. It is an approved orphan drug used primarily to treat rare autoimmune diseases such as myasthenia gravis. It belongs to a class of drugs called neonatal Fc receptor blockers, which work by lowering the levels of harmful antibodies in the body to reduce muscle weakness.
sourced from the EU Clinical Trials Register and site verification
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