Active not recruitingPHASE3INTERVENTIONAL

A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

This study is for adults with a blood condition called primary immune thrombocytopenia (ITP). ITP causes low platelet counts, which can lead to bruising and bleeding. The study is called a Phase 3 trial, which means it’s one of the final steps before a new treatment can be made widely available. Researchers are testing a new treatment, given as an injection under the skin, called efgartigimod PH20 SC. They want to find out if this treatment is safe and how well it helps improve platelet counts compared to other options. If you’ve previously taken part in a related study for this treatment, there might be an option for you to continue. The overall goal is to gather more information to help people with ITP.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

argenx

Enrolment target

173

Start

17 Nov 2021

Estimated completion

01 Oct 2026

Primary Immune Thrombocytopenia

What is this study about?

This study is designed to learn more about a new medication called efgartigimod PH20 SC. It's being tested for adults who have a condition called primary immune thrombocytopenia (ITP). ITP is a condition where your body's immune system mistakenly attacks and destroys your own platelets. Platelets are important blood cells that help your blood clot, so having too few can lead to easy bruising, bleeding, and sometimes more serious problems.

In this study, researchers want to see how well efgartigimod PH20 SC works to improve platelet counts and reduce bleeding in people with ITP. They also want to make sure the treatment is safe and doesn't cause too many side effects. This is a "Phase 3" study, which means it's one of the last stages of testing before a new medicine can potentially be approved for wider use.

The medication is given as an injection under the skin, and the study is focusing on adults. If you've previously been involved in an earlier study looking at this specific treatment for ITP, there's a possibility you might be able to continue receiving it as part of this new study. This helps gather long-term information about the treatment's effects and safety.

Key takeaways

This study is testing a new injection for adults with ITP.
It's mainly for people who have already been in a previous study for this treatment.
The main goals are to check how safe and effective the treatment is over time.
Participation involves regular clinic visits and following treatment instructions.
You would be contributing to research that could help others with ITP.

Who may be eligible?

This study is for adults aged 18 and over, of any gender. To take part, you need to understand what the study involves and be able to give your informed consent. This means you agree to participate and understand how your health information will be used for research purposes.

Crucially, this particular study is mainly for people who have already taken part in a previous study called ARGX-113-2004 and have completed their 24-week treatment period in that study. If you had any serious side effects in the previous study, the doctors and study sponsors would need to carefully review your situation to decide if it's safe for you to join this extension phase.

There are also some important rules about birth control for women who might get pregnant. You would need to use birth control methods that are in line with local guidelines, and women of childbearing age would need to have a negative pregnancy test before starting the study medication. This includes if you want to continue the treatment for another 52 weeks.

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?
Have you completed the 24-week initial period of the ARGX-113-2004 study?
Are you able to attend regular clinic appointments?
If you are a woman of childbearing potential, are you willing to use approved birth control methods?
Do you understand what the study involves and agree to take part?

Answer every question to see your result.

What does participation involve?

If you are eligible, taking part means you'll continue to receive the study medication, efgartigimod PH20 SC, which is given as an injection under the skin. You will need to attend regular visits to the study clinic. These visits are important for the doctors to monitor your health, check your platelet counts, and look for any potential side effects. They will also make sure the treatment is working as expected. If you are continuing treatment from a previous study, this additional period could last for another 52 weeks, depending on whether the medication becomes widely available. You'll need to follow the study schedule carefully, which includes taking the medication as instructed and attending all your appointments.

Potential risks and benefits

Taking part in this study could potentially offer a benefit by allowing you to continue receiving a treatment that might help manage your ITP. You would also contribute to medical knowledge, which could help others with ITP in the future. However, like all medications, efgartigimod PH20 SC can have side effects. These could be mild, like reactions at the injection site, or potentially more serious, though serious side effects are carefully monitored. Some treatments or activities are not allowed during the study, and these rules are in place for your safety. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (83)

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

Investigator Site 0010116
Verified postcode
Bentonville, United States
Investigator site 0010045
Verified postcode
Washington D.C., United States
Investigator Site 0010062
Verified postcode
Fort Wayne, United States
Investigator site US0010042
Verified postcode
Iowa City, United States
Investigator Site 0010095
Verified postcode
Oklahoma City, United States
Investigator Site 0540001
Verified postcode
Buenos Aires, Argentina
Investigator site 540004
Verified postcode
Buenos Aires, Argentina
Investigator Site 0610012
Verified postcode
Garran, Australia
Investigator Site 0610003
Verified postcode
West Perth, Australia
Investigator site 610005
Verified postcode
Westmead, Australia
Investigator Site 3590017
Verified postcode
Plovdiv, Bulgaria
Investigator site 0560003
Verified postcode
Reñaca, Chile

Common questions

What is primary immune thrombocytopenia (ITP)?

ITP is a condition where your body's immune system attacks and destroys its own platelets. Platelets are tiny blood cells needed for clotting, so low numbers can cause bruising and bleeding.

What is efgartigimod PH20 SC?

It's a new medication being tested for ITP. It's given as an injection under the skin.

Who can join this study?

This study is mainly for adults (18+) who have already participated in a specific previous study (ARGX-113-2004) and completed its first phase.

What does 'Phase 3 study' mean?

Phase 3 means it's a large study, usually one of the last steps, before a new drug might be approved for general use. It checks how safe and effective the treatment is.

What if I get pregnant during the study?

Women who can become pregnant must use effective birth control throughout the study, and a pregnancy test will be done before starting the medication.