Use of pegfilgrastim in severe chronic neutropenia
This research study is for adults with a condition called severe chronic neutropenia (SCN). SCN means you have too few neutrophils, a key type of white blood cell that helps your body fight off infections. The study is investigating a medicine called Neulasta (also known as pegfilgrastim) to see how well it can increase the number of these important infection-fighting cells in the blood over a period of several months. Researchers will also be looking at how Neulasta might affect the number, types, and length of infections you get, how many times you need antibiotics, and if it changes how often you need to stay in hospital. This is an early stage study, aiming to understand the medicine better.
At a glance
What is this study about?
This study is about a rare blood condition called severe chronic neutropenia (SCN). If you have SCN, your body doesn't make enough of a certain type of white blood cell called neutrophils. Neutrophils are incredibly important for your immune system, acting like tiny soldiers that rush to fight off germs and infections. When you don't have enough of them, you can get sick more often and infections can become serious.
The main goal of this study is to see if a medicine called Neulasta (or pegfilgrastim) can help people with SCN produce more neutrophils. We want to find out if taking Neulasta regularly over several months can lead to a stable and healthier level of these infection-fighting cells. This is important because having more neutrophils could mean you're better protected against getting sick.
Beyond just counting the cells, the researchers will also be keeping a close eye on other important things. They want to understand if Neulasta can reduce how many infections you get, how serious those infections are, and how long they last. They'll also be tracking how often people need to take antibiotics or stay in hospital because of infections. This will help them understand the full impact of Neulasta on the health and daily life of people with SCN. This is a 'Phase II' study, which means it’s an early step in testing a new treatment, aiming to learn more about its effects and safety.
Key takeaways
- This study is for adults with severe chronic neutropenia (SCN).
- It tests if Neulasta medicine can increase infection-fighting white blood cells.
- Researchers will also track infections, antibiotic use, and hospital stays.
- Participation involves regular clinic visits for blood tests and health checks.
- You can leave the study at any time without affecting your usual care.
Who may be eligible?
This study is open to adult men and women, meaning you must be at least 18 years old to take part. There is no upper age limit, so older adults are also welcome to join in.
To be considered for this study, you must have been diagnosed with severe chronic neutropenia (SCN), which is the specific condition this research is focusing on.
The medical team will carefully check your health records and perform some tests to make sure that the study is safe and right for you. They will explain everything clearly during this process.
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 18 years old or older?
- Have you been diagnosed with severe chronic neutropenia (SCN)?
- Are you able to attend regular clinic appointments for several months?
- Are you willing to receive an injection of the study medicine?
What does participation involve?
If you join this study, you will receive the study medicine, Neulasta, as an injection. The study will last for several months. During this time, you'll have regular visits to the clinic to monitor your health and how the medicine is working. These visits will likely include blood tests to check your neutrophil count and other health markers, and you'll also be asked about any infections you've experienced. You might also be asked to keep a diary of your symptoms. The exact number of visits, tests, and total duration will be fully explained by the study team, but generally, the main part of the study will involve monitoring for a period of five months after the first month of treatment.
Potential risks and benefits
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Common questions
What is severe chronic neutropenia (SCN)?
SCN is a condition where your body doesn't produce enough of a specific type of white blood cell, called neutrophils, which are crucial for fighting infections.
What is Neulasta?
Neulasta is the name of the medicine being tested in this study. It's designed to help your body make more of those important infection-fighting white blood cells.
What will researchers be looking for?
The main thing they'll check is if Neulasta increases your neutrophil count. They'll also watch for changes in your infections, antibiotic use, and hospital stays.
How long does the study last?
The main part of the study will involve monitoring over a period of five months after the first month of treatment, with regular visits during this time.
Will I have to pay to be in the study?
No, you will not have to pay for the study medication or the extra study-related visits and tests.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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