Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

ARGX-113-1803: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia

This research study is testing a new medicine, efgartigimod, for adults living with primary immune thrombocytopenia (ITP). ITP is a condition where your body attacks its own platelets, which are tiny blood cells that help stop bleeding. The main goal of this study is to understand if efgartigimod is safe to use over a long period and how well it helps increase platelet counts. Researchers will closely monitor for any side effects and track how often and how severely they occur. They will also check other health indicators like vital signs and blood test results to get a full picture of the medicine's effects. This is a "Phase 3" study, meaning it's a later stage of research to confirm the medicine's effectiveness and safety in a larger group of patients.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Argenx

Enrolment target

Start

06 May 2024

Primary immune thrombocytopenia

What is this study about?

This study is about a condition called primary immune thrombocytopenia, or ITP for short. If you have ITP, your body's immune system mistakenly attacks and destroys your platelets. Platelets are crucial for helping your blood clot, so low platelet counts can lead to bruising, bleeding, and serious health risks.

Researchers are investigating a new medicine called efgartigimod. The main aim of this study is to find out if this medicine is safe when used for a long time and how well it helps people with ITP. They will be carefully looking for any side effects and checking how often they happen and how serious they are. They will also be tracking your platelet count, along with other health measurements like your blood pressure and heart rate, through regular blood tests.

This is a "Phase 3" study, which means it's one of the final steps before a new medicine might be approved for wider use. It's designed to confirm the good results from earlier studies and gather more information on effectiveness and safety in a larger group of people. By taking part, you could help doctors learn more about ITP and potentially offer a new treatment option for others in the future.

Key takeaways

This study is testing a new medicine, efgartigimod, for adults with ITP.
It aims to check the long-term safety and effectiveness of the medicine.
The medicine is given directly into a vein.
Researchers will closely monitor platelet counts and side effects.
Participation helps scientists learn more about ITP and potential new treatments.

Who may be eligible?

This study is open to adults aged 18 years and older who have primary immune thrombocytopenia (ITP).

If you're interested, the study team will need to check some other things to make sure the study is a good fit for you and that you meet all the necessary health requirements. This helps keep you safe and ensures the study results are as clear as possible.

Both men and women can take part in this study.

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.

Are you 18 years old or older?
Do you have a diagnosis of primary immune thrombocytopenia (ITP)?
Are you able to attend regular clinic visits?
Are you comfortable receiving IV medication?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive the study medicine, efgartigimod, given directly into your vein (intravenously). You will have regular visits to the clinic where the study team will monitor your health closely. During these visits, they will check your vital signs (like blood pressure and heart rate), take blood samples to measure your platelet count and do other lab tests, and ask you about any side effects you might be experiencing. The study will look at your progress over an extended period to understand the long-term effects of the medicine. The specific number of visits and the total length of your participation will be explained in detail by the study team.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might benefit from receiving a new treatment that could improve your condition, or you could help doctors learn more about ITP, which could help future patients. However, there's always a chance of side effects from the study medicine, or that it may not work for you. The study team will explain all known risks before you decide to join. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Czechia
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Unverified
Poland
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Unverified
Hungary

Common questions

What is primary immune thrombocytopenia (ITP)?

ITP is a condition where your body's immune system mistakenly attacks and destroys your platelets, which are cells vital for blood clotting.

What is efgartigimod?

Efgartigimod is the new experimental medicine being tested in this study to see if it can help improve platelet counts in people with ITP.

What does 'intravenous' mean?

It means the medicine will be given directly into your vein using a drip, similar to how fluids or some antibiotics are given.

Why is this called a 'Phase 3' study?

Phase 3 is a late stage of research where a medicine is tested in a larger group of people to confirm its safety and how well it works.

Will I know if I'm getting the actual medicine?

This particular study is "open-label," meaning both you and the study team will know you are receiving the active study medicine, efgartigimod.