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

The research process

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

Step 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
Step 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
Step 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
Step 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 trial?

16 criteria

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
check
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
- check
  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 trial?

13 criteria

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

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Verified sites

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Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France
Responsive
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Lodz, Poland
Responsive
Semmelweis University
Budapest, Hungary

Trial locations

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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.

Efgartigimod Alfa

What is already known about the treatment

Efgartigimod - An intravenously administered antibody-based medication that is being investigated for the treatment of chronic idiopathic thrombocytopenic purpura (ITP), a condition where the immune system attacks blood platelets. This drug works by targeting and reducing the levels of immunoglobulin G (IgG) antibodies that are responsible for destroying platelets in ITP patients, thereby helping to maintain normal platelet counts. Efgartigimod belongs to the class of immunomodulating agents and is currently being studied in clinical trials for pediatric patients aged 12 to 18 years, with promising results in managing this chronic autoimmune disorder.

Investigated diseases

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.