Study 1002-041: An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic Acid in Pediatric Patients (6 to 17 Years of Age) with Heterozygous Familial Hypercholesterolemia
This research study is for children and teenagers between 6 and 17 years old who have a genetic condition called heterozygous familial hypercholesterolemia (HeFH). HeFH causes very high levels of 'bad' cholesterol from a young age. The study is looking at a medicine called bempedoic acid. Researchers want to understand exactly how this medicine behaves in the bodies of young people (how much gets into their blood and how long it stays there) and if it helps lower their cholesterol. We also want to check if the medicine is safe for them to take and if different forms of the medicine are easy for young people to swallow.
At a glance
What is this study about?
This study is focusing on a medicine called bempedoic acid for young people who have a condition called heterozygous familial hypercholesterolemia, often shortened to HeFH. This is a genetic condition inherited from a parent that causes very high levels of 'bad' cholesterol (LDL-C) in the blood, even from childhood. High cholesterol can increase the risk of heart problems later in life.
The main aim of this study is to learn more about how bempedoic acid works in children and teenagers (aged 6 to 17). We want to understand how much of the medicine gets into their bloodstream and how long it stays there over eight weeks. This helps doctors figure out the best dose and how often it should be given. We also want to see if the medicine helps to lower cholesterol levels.
Another important part of the study is to check the safety of bempedoic acid in this age group. Researchers will also look at whether different forms of the medicine (like tablets or liquids) are easy for young people to take and if they like the taste. This information is important for making sure future treatments are both effective and easy for young patients to use.
Key takeaways
- This study is for children and teenagers (6-17) with high cholesterol due to HeFH.
- It's testing a medicine called bempedoic acid to see how it works and if it's safe.
- Participation involves taking the medicine and having regular check-ups and blood tests.
- The study aims to find the best way to use this medicine for young people.
- Your involvement could help future patients with HeFH.
Who may be eligible?
This study is designed for children and teenagers. If you are aged between 6 and 17 years old, you might be able to take part. This study welcomes both boys and girls.
The most important health requirement for joining is having a specific genetic condition called heterozygous familial hypercholesterolemia, or HeFH. This means you have high cholesterol levels because of a gene you inherited from one of your parents.
There might be other health details that make someone suitable or unsuitable for the study, but these would be discussed in full by the study team if you express an interest.
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 6 and 17 years old?
- Have you been diagnosed with heterozygous familial hypercholesterolemia (HeFH)?
- Are you able to attend appointments for blood tests?
- Are you willing to take a study medicine regularly?
What does participation involve?
If you decide to take part in this study, you will be given the study medicine, bempedoic acid, to take regularly. During the study, you will have several visits to the clinic. These visits will involve blood tests to measure the amount of medicine in your body at different times, as well as to check your cholesterol levels and overall health. You might also be asked questions about how you feel and if the medicine is easy to take.
The study will involve taking the medicine steadily for 8 weeks. After this main part, there will also be follow-up checks to continue monitoring your health and the effects of the medicine. The research team will explain the exact schedule of visits and tests, and the total duration of your involvement, before you make any decisions.
Potential risks and benefits
Locations (4)
- —UnverifiedGermany
- —UnverifiedNetherlands
- —UnverifiedDenmark
- —UnverifiedSpain
Common questions
What is heterozygous familial hypercholesterolemia (HeFH)?
HeFH is a genetic condition that causes very high levels of 'bad' cholesterol (LDL-C) from a young age, increasing the risk of heart problems.
What is bempedoic acid?
Bempedoic acid is a medicine that helps to lower cholesterol levels in the blood.
Why are they studying this medicine in children?
Doctors want to learn how this medicine works and if it's safe and effective specifically for children and teenagers with HeFH, to see if it can help them manage their cholesterol.
Will I have to take medicine if I join?
Yes, if you join, you will be given bempedoic acid for a period of time as part of the study.
How long will the study participation last?
You will take the study medicine for 8 weeks, with follow-up appointments after that. The exact total duration will be explained by the study team.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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