Study of efgartigimod given by IV infusion in adolescents aged 12-17 years with chronic immune thrombocytopenia

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What is this study about?

This study focuses on young people aged 12 to 17 who have Chronic Immune Thrombocytopenia (ITP), a rare blood disorder where the body’s immune system mistakenly attacks and destroys blood platelets, leading to easy bruising and bleeding. The study will test a medication called efgartigimod, given through intravenous infusion, compared to placebo to find the right dose for young patients with this condition.

The study consists of two parts. In the first part, participants will be randomly assigned to receive either efgartigimod or placebo through regular infusions. Neither the participants nor their doctors will know which treatment they are receiving during this phase. In the second part, all participants will receive the actual medication efgartigimod.

Throughout the study, researchers will monitor how the medication moves through the body, its effects on blood components, particularly platelets and antibodies, and its overall safety. They will also track any bleeding episodes and assess how the treatment impacts the participants’ quality of life. The study will evaluate if efgartigimod can help maintain adequate platelet counts in young patients with chronic ITP.

1 Initial evaluation

Your platelet count will be checked to confirm it is less than 30×109/L

Your medical history of chronic immune thrombocytopenia (ITP) will be reviewed to confirm it has lasted more than 12 months

If you are a female who can become pregnant, a pregnancy test will be performed

2 Double-blind treatment period

You will be randomly assigned to receive either efgartigimod or placebo through intravenous (IV) infusion

Neither you nor your doctor will know which treatment you are receiving

Your platelet count will be monitored regularly

Blood samples will be taken to measure the medication levels and immune system response

Your bleeding symptoms will be assessed using a special scoring system

3 Open-label treatment period

If you received placebo during the first part, you will now receive efgartigimod

This period lasts 24 weeks

Regular monitoring of platelet counts continues

Your response to treatment will be evaluated by checking if your platelet count reaches 50×109/L or higher

4 Health assessments

Throughout the study, you will complete questionnaires about your quality of life and fatigue levels

Regular safety checks will include blood tests, heart monitoring (ECG), and vital signs

Any side effects will be recorded and monitored

Who Can Join the Study?

  • Must be between 12 and 17 years old at the time of giving consent, with parent/guardian approval
  • Must be able to understand and follow study requirements
  • Must have a diagnosis of primary ITP (a condition where blood platelet count is low) for more than 12 months
  • Must have a platelet count below 30,000 per microliter of blood before starting the study
  • Must have received previous treatment with at least one of these medications:
    • Corticosteroids (anti-inflammatory medications)
    • IVIg (antibodies given through a vein)
    • Anti-D immunoglobulin (for certain eligible patients)
    • TPO-RAs (medications that help produce platelets)
    • Rituximab (a medication that affects the immune system)
  • Must have shown some response to at least one previous treatment, achieving a platelet count of at least 50,000 per microliter
  • Must have documented insufficient response to previous treatments, meaning the treatment stopped working or didn’t work well enough
  • Female participants who can become pregnant must:
    • Use approved contraception methods
    • Have negative pregnancy tests before starting the study
  • Must have several documented platelet count measurements:
    • At least 3 measurements in the past 3 months
    • At least 2 measurements in the 14 days before starting
    • One measurement on the first day of the study

Who Cannot Join the Study?

  • History of platelet transfusion (transfer of blood platelets from donor) within 2 weeks before starting the study
  • Previous treatment with rituximab (a medication that affects immune system) within 6 months before the study
  • Active bacterial infection requiring treatment with antibiotics
  • Known active viral infection including hepatitis B, hepatitis C, or HIV
  • History of blood clots or thrombotic events in the past 12 months
  • Major surgery within 4 weeks before starting the study
  • Pregnancy or breastfeeding
  • Current participation in other clinical trials
  • History of autoimmune conditions (diseases where immune system attacks healthy cells) other than ITP
  • Significant heart, liver, or kidney disease
  • History of cancer within the past 5 years
  • Use of prohibited medications within specified timeframes before study start
  • Known allergic reactions to study medication or similar substances

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital Universitario 12 De Octubre Madrid Spain
Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos Kaunas Lithuania
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Uniwersytecki Szpital Dzieciecy W Krakowie Cracow Poland
Samodzielny Publiczny Dzieciecy Szpital Kliniczny Im. Jozefa Polikarpa Brudzinskiego W Warszawie Warsaw Poland
University Of Debrecen Debrecen Hungary
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Vilniaus universiteto ligonine Santaros klinikos VšĮ Vilnius Lithuania
Semmelweis University Budapest Hungary
Borsod-Abauj-Zemplen Varmegyei Koezponti Korhaz Es Egyetemi Oktatokorhaz Miskolc Hungary
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Fundeni Clinical Institute Bucharest Romania
Uhxkdhgnhz Hbngjvav Ctyyfdf Cologne Germany
Afnatdz Ouyzqwuprlq Unzvgqbgnopoa Clabdqvhyyrd Dvngi Srjwnh E Dzbhj Sbktasj Dq Tegyqc Turin Italy
Uqjmgebpjqebse Cdhvpyw Khnfiavms Gdansk Poland
Hiekcvqd Vcuw djrbrltk Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
05.09.2025
Germany Germany
Recruiting
05.09.2025
Hungary Hungary
Not yet recruiting
05.09.2025
Italy Italy
Recruiting
05.09.2025
Lithuania Lithuania
Recruiting
05.09.2025
Poland Poland
Recruiting
05.09.2025
Romania Romania
Recruiting
05.09.2025
Spain Spain
Recruiting
05.09.2025

Trial locations

Investigated drugs:

Efgartigimod IV is a medication designed to treat chronic immune thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys platelets in the blood. This medication works by reducing the levels of antibodies that attack platelets, which may help increase platelet counts in patients. It is administered through intravenous (IV) infusion, meaning it is given directly into a vein.

Chronic Idiopathic Thrombocytopenic Purpura – A blood disorder where the immune system mistakenly attacks and destroys platelets, which are blood cells needed for normal blood clotting. This condition develops gradually and persists for a long time, with the body’s immune system, particularly antibodies, targeting platelets as if they were harmful substances. The reduced number of platelets in the blood can lead to easy bruising and bleeding. The term “idiopathic” means that the exact cause of the condition is unknown. This condition can affect people of any age, though it’s more common in certain age groups, and may fluctuate in severity over time.

Trial ID:
2025-521055-23-00
Protocol code:
ARGX-113-2409
Trial Phase:
Therapeutic use (Phase IV)

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