Not recruiting

Long-term Study on the Safety and Efficacy of Efgartigimod in Adults with Primary Immune Thrombocytopenia

Verified siteInvestigationalNo placebo

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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 an autoimmune disorder where the body's immune system mistakenly attacks and destroys its own platelets, which are blood cells that help with clotting. This can lead to easy or excessive bruising and bleeding. The treatment being tested in this study is called efgartigimod, also known by its code name ARGX-113. Efgartigimod is given as a solution for infusion, which means it is administered directly into the bloodstream through a vein.

The purpose of this study is to evaluate the long-term safety of efgartigimod in adult patients with primary immune thrombocytopenia. Participants in the study will receive efgartigimod infusions and will be monitored over a period of time to assess how safe the treatment is and how well it works in managing the condition. The study will involve regular visits to the clinic for infusions and check-ups, where doctors will keep track of the participants' health and any changes in their platelet counts.

Throughout the study, researchers will collect information on the frequency and severity of any side effects, as well as changes in vital signs and laboratory test results. The study aims to provide valuable insights into the long-term use of efgartigimod for treating primary immune thrombocytopenia, helping to determine if it is a safe and effective option for patients with this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the trial
Upon joining the trial, the patient must have completed a previous 24-week trial period. Eligibility includes understanding the trial requirements and providing written consent.
Women of childbearing potential must have a negative pregnancy test before starting the trial medication.
Step 2
Initial treatment phase
The patient receives the medication efgartigimod, also known as ARGX-113, administered as a solution for infusion through intravenous use.
The dosage is 10 mg per kg of body weight. The frequency and duration of administration are determined by the trial protocol.
Step 3
First 52-week treatment period
During this period, the primary focus is on monitoring the safety of the medication. This includes tracking any adverse effects, changes in vital signs, and laboratory assessments.
Secondary objectives include evaluating the control of the disease by measuring platelet counts and assessing the need for additional treatments.
Step 4
Additional 52-week treatment period
If the medication is not yet commercially available, the patient may continue treatment for an additional 52 weeks, provided they understand and consent to this extension.
The focus remains on safety and efficacy, with continued monitoring of platelet counts and any potential side effects.
Step 5
End of trial
The trial is estimated to conclude by March 2026. At the end of the trial, all collected data will be analyzed to assess the long-term safety and efficacy of the medication.

Who can join the trial?

6 criteria

Must be able to understand the trial requirements and agree to participate by signing a consent form. This includes agreeing to share health information related to the research and following the trial procedures, such as attending required visits.
Must have participated in a previous trial called ARGX-113-1801 and completed the 24-week trial period.
Women who can have children must have a negative pregnancy test before starting the trial medication. Women are considered able to have children unless they have not had a period for at least a year and have a specific hormone level, or have had surgery to prevent pregnancy.
Women who can have children should use a reliable method of birth control during the trial and for 90 days after the last dose of the trial medication. This includes methods like hormonal birth control, an intrauterine device (IUD), or having a partner who has had a vasectomy. Abstaining from sex is only acceptable if it is the person's usual lifestyle.
If the trial medication is not available for purchase or through another program, participants must understand and agree to an additional 52-week treatment period, including signing a consent form and following trial procedures.
Must have completed a 52-week treatment period.

Who cannot join the trial?

3 criteria

Patients who have a different condition than primary immune thrombocytopenia cannot participate. This is a condition where the immune system attacks and destroys platelets, which are cells that help blood clot.
Patients who are not adults cannot participate. This means only individuals who are 18 years or older are eligible.
Patients who are not able to give their own consent or understand the study cannot participate. This includes individuals who might be considered part of a vulnerable population.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Azienda Ospedaliera Universitaria Senese
Siena, Italy
Verified
Azienda Ospedaliero-Universitaria Maggiore Della Carita
Novara, Italy
Verified
Azienda USL IRCCS Di Reggio Emilia
Reggio Emilia, Italy

Trial locations

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Investigated drugs

Efgartigimod (ARGX-113) is a medication being studied for its safety and effectiveness in treating adults with primary immune thrombocytopenia (ITP). It is administered through an intravenous infusion. The goal of the trial is to understand how well patients tolerate the medication over a long period and to see how it affects their condition.

Efgartigimod Alfa

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 safety and effectiveness in treating primary immune thrombocytopenia, a condition where the immune system mistakenly attacks and destroys platelets in the blood. Efgartigimod works by targeting and reducing the activity of certain antibodies that contribute to this immune response. It is classified as an immunomodulatory agent, which means it helps to regulate or normalize the immune system's activity.

Investigated diseases

Primary Immune Thrombocytopenia – 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 essential for normal blood clotting. In many cases, the exact cause of this immune response is unknown. The disease can progress with varying degrees of severity, with some individuals experiencing mild symptoms and others having more significant bleeding issues. Over time, the platelet count may fluctuate, leading to periods of remission and relapse. The progression of the disease can be unpredictable, with some patients maintaining stable platelet levels while others may experience a decline.