A Phase 3, Multicenter, Open-Label, Long-Term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is an autoimmune condition where the body's immune system attacks and destroys its own platelets. Platelets are tiny blood cells that help blood clot. Having too few platelets can cause bruising, nosebleeds, and more serious bleeding. This clinical study is testing a drug called efgartigimod (also known as ARGX-113) in adults with ITP. The medicine is given as an injection just under the skin. It aims to reduce the harmful proteins that attack platelets. The main goal of this trial is to check the long-term safety of the medicine and see if it causes any unwanted side effects. Doctors will also track whether the drug helps raise platelet counts and keeps them stable, potentially reducing the need for other ITP treatments. Everyone taking part receives the active drug.
At a glance
What is this study about?
Primary immune thrombocytopenia, or ITP, is a rare blood condition. Normally, your immune system creates proteins called antibodies to fight off bugs and infections. In people with ITP, the immune system mistakenly makes antibodies that destroy healthy platelets. Platelets are crucial because they plug cuts and stop bleeding. When platelet levels drop too low, simple knocks can cause large bruises, and cuts may bleed for a long time.
This study is testing an investigational medicine called efgartigimod. It is designed to lower the specific antibodies that attack platelets, giving the body a chance to rebuild and maintain its platelet levels. In this study, the medicine is given as a simple injection just under the skin (subcutaneous injection), combined with a substance that helps the body absorb it smoothly.
This trial is an 'open-label' study, which means there is no dummy drug (placebo). Every participant receives the real medicine. Researchers want to learn how safe the treatment is over a longer period, what side effects might happen, and how well it helps control bleeding risks in everyday life. Finding new ways to manage ITP is important, as current treatments do not work for everyone or can cause challenging side effects.
Key takeaways
- The trial tests efgartigimod, given as an injection under the skin, for adults with primary ITP.
- The goal is to test the drug's long-term safety and see if it keeps platelet counts at a safe level.
- This is an open-label study, so all participants receive the active medicine.
- Taking part involves regular clinic visits, blood tests, and heart monitoring for at least a year.
- Participation is entirely voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is open to adults aged 18 and over who have been diagnosed with primary immune thrombocytopenia (ITP). Both men and women can take part.
To join, participants must have low platelet levels that need treatment, or they may be moving directly into this study after finishing an earlier connected trial for the same medicine.
People with other medical conditions that cause low platelets, or those with certain severe heart, kidney, or liver problems, may not be able to join. The study doctors will run blood tests and health checks to make sure the trial is suitable and safe for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have a confirmed diagnosis of primary immune thrombocytopenia (ITP).
- My blood platelet counts are lower than normal.
- I am willing to attend regular hospital appointments for check-ups and blood tests.
- I am happy to receive treatment via an injection under the skin.
What does participation involve?
If you take part, you will receive efgartigimod as an injection under the skin on a regular schedule. The main part of the study lasts for at least 52 weeks (one year), with regular follow-up visits to see how you are doing.
During hospital or clinic visits, the study team will check your vital signs (such as blood pressure and pulse), run heart checks (ECGs), and take regular blood samples to measure your platelet levels. You will also be asked about any symptoms or side effects you experience.
If the treatment helps raise your platelets, the study team may also see if it is safe to reduce any other ITP medicines you are taking. You will have regular contact with specialist doctors throughout the trial to closely monitor your health.
Potential risks and benefits
Locations (6)
- —UnverifiedItaly
- —UnverifiedRomania
- —UnverifiedGreece
- —UnverifiedBulgaria
- —UnverifiedPortugal
- —UnverifiedNorway
Common questions
Will I definitely receive the real medicine?
Yes. This is an open-label study, meaning there is no dummy treatment (placebo). Every person who takes part receives efgartigimod.
How is the study drug given?
The medicine is given as an injection just under the skin (subcutaneously), similar to how insulin is given, rather than through a slow drip into a vein.
Can I keep taking my current ITP medications?
Many participants continue their usual ITP treatments when starting the trial. If your platelet count improves, your study doctor may carefully lower your other doses.
Does taking part cost anything?
No. The trial medication, clinic visits, and all study-related tests are provided free of charge, and travel expenses are often reimbursed.
What happens if I want to stop the trial?
You are free to leave the trial at any point. Your doctors will simply discuss other standard treatment options to keep managing your ITP safely.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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