Not recruitingRare disease

Safety and Efficacy Study of Efgartigimod PH20 Subcutaneous in Adults with Primary Immune Thrombocytopenia

Fast replyInvestigationalNo 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), which is a disorder that can lead to easy or excessive bruising and bleeding due to low levels of platelets in the blood. The treatment being tested in this study is called efgartigimod, also known by its code name ARGX-113. This medication is administered as a solution for injection under the skin, which is referred to as subcutaneous injection.

The purpose of the study is to evaluate the safety and effectiveness of efgartigimod in adult patients with Primary Immune Thrombocytopenia. Participants in the study will receive the treatment over a long-term period, and the study will monitor how well the treatment works and how safe it is for the patients. The study will also look at how the treatment affects platelet counts, which are important for blood clotting, and will track any side effects or adverse events that may occur during the treatment period.

Throughout the study, participants will have regular visits to check their health and monitor their response to the treatment. The study aims to provide valuable information on the potential benefits and risks of using efgartigimod for treating Primary Immune Thrombocytopenia, helping to determine if it can be 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 participant must have completed a previous 24-week trial period. Eligibility is assessed on a case-by-case basis, especially if any serious adverse events occurred during the previous trial.
Participants must agree to use contraceptive measures and, if applicable, provide a negative pregnancy test before starting the trial.
Step 2
Initial treatment phase
The trial involves the administration of efgartigimod alfa, known as ARGX-113, which is a solution for injection.
The primary goal is to evaluate the safety and tolerability of this medication in adult patients with primary immune thrombocytopenia (ITP).
Step 3
First 52-week treatment period
During the first 52 weeks, the participant's platelet counts are monitored to assess the extent of disease control.
The participant may receive training for self-administration of the medication at home, and the number of training visits required will be recorded.
The trial will also track the incidence and severity of any adverse events, as well as changes in quality of life and fatigue levels.
Step 4
Additional 52-week treatment period
If the medication is not commercially available, participants may continue treatment for an additional 52 weeks.
The same monitoring and assessments from the first 52-week period will continue, including the presence of antibodies against the medication.
Step 5
End of trial
The trial is estimated to conclude by December 13, 2026.
Final assessments will include a review of all collected data on safety, efficacy, and any adverse events experienced during the trial.

Who can join the trial?

7 criteria

Must be an adult with Primary Immune Thrombocytopenia (a condition where the immune system attacks and destroys platelets, which are cells that help blood clot).
Must be able to understand the trial requirements and provide written consent, which means agreeing to participate and allowing the use of health information for research.
Must be willing and able to follow the trial procedures, including attending all required visits.
If previously participated in the ARGX-113-2004 trial and completed the 24-week period, eligibility will be checked, especially if any serious side effects occurred during that trial.
Must agree to use birth control methods as per local rules. Female participants who can have children must have a negative pregnancy test before starting the trial medication.
If continuing with the trial for an additional 52 weeks, must understand and agree to the requirements of this extended period, including providing written consent and attending all required visits.
Must have completed a 52-week treatment period if continuing with the trial.

Who cannot join the trial?

6 criteria

Patients who have a different condition than Primary Immune Thrombocytopenia cannot participate. This condition is a disorder where the immune system attacks and destroys platelets, which are cells that help blood clot.
Patients who are not adults are excluded. This means only individuals who are 18 years or older can participate.
Patients who are not able to understand or follow the study procedures will be excluded. This means participants need to be able to understand what the study involves and follow instructions.
Patients who have any other medical condition that the study doctors think might make it unsafe for them to participate will be excluded. This means if there is any health concern that could be risky, the patient cannot join the study.
Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
Patients who are currently participating in another clinical trial will be excluded. This is to avoid any interference with the results of either study.

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Verified sites

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Azienda Ospedaliera Universitaria Federico II Di Napoli
Verified
Naples, Italy
Sykehuset Oestfold HF Kalnes
Responsive
Graalum, Norway
University Multiprofile Hospital For Active Treatment Saint Georgi EAD
Responsive
Plovdiv, Bulgaria

Trial locations

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Investigated drugs

Efgartigimod (ARGX-113) PH20 Subcutaneous is a medication being studied for its safety and effectiveness in treating adults with primary immune thrombocytopenia (ITP). This condition is characterized by a low platelet count, which can lead to easy or excessive bruising and bleeding. The medication is administered under the skin and is designed to help increase platelet counts by modulating the immune system.

Efgartigimod Alfa

What is already known about the treatment

Efgartigimod – Efgartigimod is administered subcutaneously, meaning it is injected under the skin. It is currently being studied in clinical trials for its safety and effectiveness in treating primary immune thrombocytopenia, a condition where the immune system attacks and destroys platelets. The medication works by targeting and reducing the activity of certain antibodies that contribute to the disease. Efgartigimod is classified as an immunomodulatory agent, which means it helps to regulate or normalize the immune system's function.

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. It can develop suddenly or gradually and may be triggered by infections or other immune disorders. Symptoms often include petechiae, which are small red or purple spots on the skin, and prolonged bleeding from cuts. The severity of the condition can vary, 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.