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Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, 52-week, open-label, single arm study to investigate the efficacy and safety of mepolizumab SC in participants aged 6 to 17 years with hypereosinophilic syndrome.

This research study is looking at a condition called hypereosinophilic syndrome (HES) in children and teenagers aged 6 to 17. HES is a rare blood disorder where a type of white blood cell, called eosinophils, becomes too high, causing inflammation and damage to organs, leading to symptom 'flares'. The study aims to find out how well a medication called mepolizumab works to reduce these symptom flares over 52 weeks. It will also check if mepolizumab is safe and how it might affect the amount of steroid medicine people need to take for their HES. This is an important step to see if this treatment could help young people manage their HES better.

At a glance

Status
Ongoing, recruiting
Phase
Therapeutic confirmatory (Phase III)
Sponsor
Glaxosmithkline Research & Development Limited
Enrolment target
7
Start
01 Jul 2024

What is this study about?

This study is about a health condition called hypereosinophilic syndrome, or HES. HES is a rare disorder that happens when your body makes too many special white blood cells called eosinophils. These extra cells can travel around the body and cause problems, leading to a variety of symptoms known as 'flares'. These flares can be very uncomfortable and affect daily life.

The main goal of this study is to see if a medication called mepolizumab can help reduce how often these HES flares happen in young people aged 6 to 17. Researchers will be looking closely at flare-ups over a year (52 weeks) to see if the medicine makes a difference. They also want to understand if mepolizumab is a safe treatment for children and teenagers with HES.

Another important part of the study is to see if mepolizumab can help people reduce the amount of steroid medicine they need to take. Steroids are often used to treat HES, but they can have side effects when used long-term. If mepolizumab helps lower the need for steroids, it could improve the health and well-being of young people with HES. The study will also look at how fatigue is affected and generally monitor the body's response to the drug.

Key takeaways

  • This study is for young people (ages 6-17) with HES.
  • It aims to see if mepolizumab can reduce HES flare-ups over a year.
  • Researchers will also check if the medicine is safe and can reduce steroid use.
  • Participation involves regular clinic visits and medication injections.
  • You can stop participating at any time.
  • Discuss all questions and concerns with your doctor.

Who may be eligible?

This study is specifically for children and teenagers who have been diagnosed with hypereosinophilic syndrome (HES).

Participants must be between the ages of 6 and 17 years old to be considered for this research. Both boys and girls can take part.

When you talk to the study doctor, they will review your medical history and current health to make sure this study is the right fit for you and that you meet all the necessary requirements to participate safely.

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.

  1. I am between 6 and 17 years old.
  2. I have been diagnosed with hypereosinophilic syndrome (HES).
  3. I am able to attend regular clinic visits for about a year.
  4. I understand that the study involves injections of medicine under the skin.
  5. My doctor has confirmed that there are no medical reasons preventing me from joining.
Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the study medication, mepolizumab, as an injection under the skin. This will happen regularly over a period of 52 weeks (about one year).

Throughout the year, you will have regular visits to the clinic so the study team can check on your health, monitor your HES symptoms and flares, and see how you are responding to the medication. They will likely take blood samples to check your eosinophil levels and the amount of study medicine in your body. They will also ask about your fatigue and any other symptoms you might be experiencing.

These visits are important to make sure you are safe and that the study is collecting all the necessary information. The study team will explain the full schedule of visits and what each visit will involve.

Potential risks and benefits

Participating in a study like this may offer potential benefits, such as access to a new treatment for HES that might help reduce your symptoms and flare-ups, and possibly reduce your need for steroids. However, there are also potential risks; medicines can have side effects, and mepolizumab is no different. The study team will explain all known side effects. It's important to remember that joining a study is always your choice, and you have the right to withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    Spain
  • Unverified
    Netherlands

Common questions

What is hypereosinophilic syndrome (HES)?

HES is a rare blood condition where your body produces too many of a certain type of white blood cell called eosinophils. These extra cells can cause problems in different parts of your body.

What is mepolizumab?

Mepolizumab is the study medicine being tested. It's designed to reduce the number of eosinophils in your body, hoping to lessen HES symptoms and flares.

How will I receive the study medicine?

The medicine will be given through an injection under your skin, similar to a routine shot you might receive at the doctor's office.

How long will the study last for me?

If you join, you will be part of the study for about 52 weeks, which is one year, during which you'll receive the study medicine and have regular check-ups.

Can I stop participating in the study if I want to?

Yes, absolutely. You have the right to leave the study at any time, for any reason, and this decision will not affect your ongoing medical care.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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