A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
This research study is for young people between 12 and 17 years old who have a long-lasting blood condition called chronic Immune Thrombocytopenia (ITP). ITP causes a low number of platelets, which are important for blood clotting. The study wants to find the best dose of a new medicine, efgartigimod, given through a drip, to help manage ITP. Participants will first receive either the study medicine or a dummy medicine (placebo) for up to 24 weeks without knowing which one they have. Afterwards, everyone will receive the study medicine for at least one year, and potentially a second year. The whole study could last up to 138 weeks. This study is important to see if efgartigimod can be a safe and effective treatment option for young people with ITP.
At a glance
What is this study about?
This study is looking into a new medicine called efgartigimod for young people aged 12 to 17 who have a type of chronic Immune Thrombocytopenia (ITP). ITP is a condition where the body's immune system mistakenly attacks and destroys its own platelets, which are tiny blood cells that help stop bleeding. 'Chronic' means the condition has been present for a long time. The main goal of this particular study is to work out the most effective dose of efgartigimod, given as an intravenous (IV) drip, to help these young people.
Finding the right dose is really important because it ensures the medicine is both safe and works as well as it can. By focusing on young people, researchers hope to provide a new treatment option specifically tailored for this age group, who sometimes don't respond well to existing treatments or need other options. The study also aims to understand how efgartigimod works in the body and if it can help increase platelet numbers to a healthier level, reducing the risk of bleeding.
Ultimately, the findings from this study could lead to efgartigimod becoming a widely available and approved treatment for young people living with chronic ITP, potentially improving their quality of life and reducing the impact of the condition. It’s part of a larger effort to find better ways to manage ITP for everyone affected.
Key takeaways
- This study is testing a new medicine, efgartigimod, for young people (12-17 years old) with long-term ITP.
- It aims to find the right dose for this age group to help increase platelet counts.
- Participants will first receive either the study medicine or a dummy treatment, then all will receive the study medicine.
- The study involves regular visits, IV treatments, blood tests, and could last over two years.
- This research hopes to provide a new treatment option for young people with ITP.
- You can withdraw from the study at any time if you decide it's not for you.
Who may be eligible?
This study is looking for young people aged between 12 and 17 years old who have been diagnosed with a type of ITP called 'primary ITP' for more than 12 months. Your doctor would have already tried to treat your ITP with at least one common treatment like steroids, IVIg, anti-D immunoglobulin, or other similar medicines, but these treatments haven't fully helped.
You should have shown some improvement to at least one of these past treatments (meaning your platelet count went up), but currently, your average platelet count needs to be quite low (less than 30 x10^9/L).
However, you can't join the study if your ITP is caused by another medical condition (this is called 'secondary ITP'), or if you have a different type of low platelet count not related to the immune system. You also wouldn't be able to take part if you have a history of serious or critical bleeding due to ITP, or if you have a family history of inherited low platelets.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 12 and 17 years old?
- Have you been diagnosed with primary ITP for over 12 months?
- Have you tried at least one ITP treatment (like steroids or IVIg) before?
- Did your platelet count improve after one of those past treatments?
- Is your current average platelet count less than 30 x10^9/L (meaning quite low)?
- Do you NOT have ITP due to another medical condition or a family history of inherited low platelets?
What does participation involve?
Taking part in this study involves several stages over a long period, potentially up to 138 weeks (about 2 years and 7 months). Initially, you'll be part of a 'double-blind' stage for up to 24 weeks. This means neither you, your family, nor your study doctors will know if you're receiving the study medicine (efgartigimod) or a dummy medicine (placebo) given through an IV drip. Everyone has a 2-in-3 chance of getting the active drug during this time.
After this, everyone who continues in the study will definitely receive efgartigimod through an IV drip during an 'open-label' period for at least one year. There's an option to continue for a second year if you and your doctor agree. Throughout the study, you'll have regular visits to the clinic for check-ups, blood tests (to measure your platelet count and overall health), and to receive your treatment. There will also be a follow-up period of about 8 weeks after you stop taking the study medicine to ensure everything is okay. Detailed information about the schedule of visits and tests will be provided by the study team.
Potential risks and benefits
Locations (9)
- Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)Verified postcodeBerlin, Germany· Recruiting
- Gaslini Children's HospitalVerified postcodeGenoa, Italy· Recruiting
- Uniwersytecki Szpital Dzieciecy w LublinieVerified postcodeLublin, Poland· Recruiting
- Institutul Clinic FundeniVerified postcodeBucharest, Romania· Recruiting
- Hospital Universitari Vall d'HebronVerified postcodeBarcelona, Spain· Recruiting
- Hospital Sant Joan de Deu BarcelonaVerified postcodeEsplugues de Llobregat, Spain· Recruiting
- Hospital Infantil Universitario Nino Jesus (HIUNJS)Verified postcodeMadrid, Spain· Recruiting
- Hospital Materno-Infantil Universitario Gregorio MaranonVerified postcodeMadrid, Spain· Recruiting
- Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)Verified postcodeCardiff, United Kingdom· Recruiting
Common questions
What is ITP?
ITP (Immune Thrombocytopenia) is a blood disorder where your body's immune system attacks and destroys its own platelets, which are essential for blood clotting. This can lead to bruising and bleeding.
What is efgartigimod?
Efgartigimod is an investigational medicine being tested to see if it can help increase platelet counts in people with chronic ITP. It's given as an IV (intravenous) drip.
What is a 'placebo'?
A placebo is a substance that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers understand if the benefits observed are truly due to the new medicine.
How long will I be in the study?
The study could last for up to 138 weeks, or about 2 and a half years, including different treatment periods and a follow-up stage.
Can I stop participating in the study if I want to?
Yes, you can choose to leave the study at any time, for any reason, without it affecting your future medical care.
How to find out more
Sabine Coppieters, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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