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Study on the Effectiveness and Safety of Efgartigimod IV for Adults with Primary Immune Thrombocytopenia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Primary Immune Thrombocytopenia (ITP). ITP is a disorder that can lead to easy or excessive bruising and bleeding, resulting from unusually low levels of platelets, which are cells that help blood clot. The study will evaluate a treatment called efgartigimod, which is given through an intravenous (IV) infusion. Efgartigimod is designed to help manage the symptoms of ITP by potentially increasing platelet counts in the blood.

The purpose of this study is to assess how effective and safe efgartigimod is compared to a placebo. Participants in the study will be randomly assigned to receive either efgartigimod or a placebo, which is a substance with no active medication. The study will begin with a phase where neither the participants nor the researchers know who is receiving the actual treatment or the placebo. This is followed by an open-label phase where all participants will receive efgartigimod. The study will monitor participants over a period to see how well the treatment controls the disease by measuring platelet counts and observing any side effects.

Throughout the study, participants will have regular visits to check their platelet levels and overall health. The goal is to determine if efgartigimod can help maintain platelet counts at a safe level, reducing the risk of bleeding and improving the quality of life for those with ITP. The study will also look at how long it takes for the treatment to start working and how long the effects last. This research aims to provide valuable information on a new potential treatment option for people living with Primary Immune Thrombocytopenia.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and initial treatment

    Upon joining the study, participants are randomly assigned to receive either efgartigimod or a placebo. This process is double-blinded, meaning neither the participant nor the study team knows which treatment is being administered.

    The treatment is administered through an intravenous infusion. The specific product used is Vyvgart 20 mg/mL concentrate for solution for infusion.

  2. Step 2

    Double-blinded treatment period

    During this period, the effectiveness of efgartigimod is compared to the placebo. The main goal is to assess the control of the disease by measuring platelet counts.

    Participants receive regular infusions and attend scheduled study visits to monitor their platelet levels and overall health.

  3. Step 3

    Open-label treatment period

    After the double-blinded period, all participants enter an open-label phase where they receive efgartigimod.

    This phase allows for further evaluation of the treatment's long-term safety and effectiveness.

  4. Step 4

    Monitoring and follow-up

    Throughout the study, participants undergo regular health assessments, including blood tests to measure platelet counts and other safety evaluations.

    The study aims to track the incidence of any side effects and the overall response to the treatment over time.

Who can join the trial?

6 criteria

  • The patient must be at least 18 years old and meet the local legal age for participating in clinical studies when signing the informed consent form.
  • The patient must have a documented average platelet count of less than 30 x 109/L before being randomly assigned to a group in the study. Platelets are small blood cells that help with clotting.
  • The patient must have had primary immune thrombocytopenia (ITP) for more than 12 months by the time they sign the informed consent form. Primary immune thrombocytopenia (ITP) is a condition where the immune system attacks and destroys platelets.
  • The patient must have previously received at least one treatment for ITP, such as corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive), thrombopoietin receptor agonists (TPO-RAs), or rituximab.
  • The patient must have shown an insufficient response to a previous ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive), TPO-RAs, or rituximab.
  • The patient must have had a previous response, defined as at least one platelet count of 50 x 109/L or higher, to at least one ITP treatment in the three years before signing the informed consent form. These treatments could include prednisone, dexamethasone, other or unspecified corticosteroids, IVIg, or anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive).

Who cannot join the trial?

5 criteria

  • Patients who have a different condition than Primary Immune Thrombocytopenia (ITP) cannot participate. ITP is a condition where the immune system attacks and destroys platelets, which are cells that help blood to clot.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
  • Patients who are not part of the specified clinical trial group cannot participate. This refers to specific characteristics or conditions that the study is focusing on.
  • Both male and female patients are eligible, so gender is not an exclusion criterion.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups like children, pregnant women, or those unable to give consent.
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Investigated drugs

Efgartigimod IV is a medication being studied for its ability to help control a condition called primary immune thrombocytopenia in adults. This condition involves a low number of platelets in the blood, which can lead to easy bruising and bleeding. The medication is given through an intravenous (IV) infusion, meaning it is administered directly into the bloodstream through a vein. The study aims to determine how well this medication works in managing the disease compared to not receiving the active treatment.

What is already known about the treatment

Efgartigimod – This medication is administered intravenously, meaning it is given directly into a vein. It is currently being studied in clinical trials for its effectiveness and safety in treating primary immune thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys platelets. Efgartigimod works by reducing the activity of certain antibodies that contribute to the disease, helping to increase platelet counts. It is classified as an immunomodulatory agent, which means it helps to regulate or normalize the immune system's function.

Investigated diseases

Primary Immune Thrombocytopenia (ITP) – This is a disorder characterized by a low platelet count, which can lead to easy or excessive bruising and bleeding. The condition occurs when the immune system mistakenly attacks and destroys platelets, which are necessary for normal blood clotting. ITP can develop suddenly or gradually and may be persistent. Symptoms can vary from mild to severe and may include petechiae, which are small red or purple spots on the skin, and bleeding from the gums or nose. The progression of the disease can be unpredictable, with periods of remission and relapse. It is considered a rare disease and can affect both children and adults.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515451-38-00Protocol codeARGX-113-2402Estimated enrolment69 patientsSponsorArgenx

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