A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
This research study is investigating a new oral medication, enlicitide decanoate, for children and teenagers between 6 and 17 years old who have a genetic condition called Heterozygous Familial Hypercholesterolemia (HeFH). People with HeFH have very high cholesterol, specifically a type called 'bad' cholesterol (LDL-C), which can increase health risks. The main goals of this study are to find out if this new medicine is safe for young people, how their bodies handle it, and if it's effective at lowering their cholesterol levels compared to a dummy pill (placebo). We want to ensure any new treatments are well-tolerated and make a real difference for children living with this condition.
At a glance
What is this study about?
This research study is looking into a new oral medication called enlicitide decanoate. It's designed for children and teenagers aged 6 to 17 who have a condition called Heterozygous Familial Hypercholesterolemia, often shortened to HeFH. HeFH is a genetic condition that means the body has trouble removing 'bad' cholesterol (known as LDL-C) from the blood, leading to very high levels. High LDL-C can be a concern for long-term health.
The main purpose of this study is to explore if enlicitide decanoate is a safe and effective treatment for these young people. We want to understand how their bodies respond to the medicine, including how it's handled over time and if it helps to lower their cholesterol more than a dummy pill (placebo). Gathering this information is really important because it helps us learn if this new medicine could be a good option for managing HeFH in children and teenagers.
By taking part, children and their families would be contributing to vital medical research that could potentially improve treatments for many others living with HeFH in the future. We are committed to making sure the study is conducted with the utmost care for participants' well-being.
Key takeaways
- This study is for children and teenagers (6-17) with high cholesterol due to HeFH.
- It's testing a new oral medicine called enlicitide decanoate.
- We want to learn if it's safe and effective at lowering 'bad' cholesterol.
- Participants will receive either the new medicine or a dummy pill (placebo).
- Regular health checks and blood tests will be part of the study.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is looking for children and teenagers between 6 and 17 years old. To take part, they must have a confirmed or suspected diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH), either through family history, clinical assessment, or a genetic test. They also need to have consistently high levels of 'bad' cholesterol (LDL-C), even while on their current treatments.
Participants should already be taking cholesterol-lowering medication like statins (and possibly other similar medicines), and their doses should have been stable for at least a month before starting the study. If they can't take statins because of side effects or personal preference, they might still be able to join if they're on other cholesterol-lowering medication.
There are some reasons why someone might not be able to join. For example, if they have a more severe form of Familial Hypercholesterolemia (called homozygous FH), have certain kidney problems (nephrotic syndrome), or have conditions that stop the body from properly absorbing food. Also, children who have recently used or are currently using certain other specific cholesterol-lowering medicines, especially a type called PCSK9 inhibitors, might not be eligible unless there's been enough time since their last dose.
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.
- Is your child between 6 and 17 years old?
- Does your child have a diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)?
- Does your child have high 'bad' cholesterol (LDL-C) levels?
- Is your child currently taking cholesterol-lowering medication (e.g., statins) or unable to take them?
- Has your child been on a stable dose of their current cholesterol medicine for at least a month?
- Has your child NOT recently had treatment with certain other specific cholesterol-lowering drugs (like PCSK9 inhibitors)?
What does participation involve?
If you decide to take part, your child would be given either the new medicine (enlicitide decanoate) or a dummy pill (placebo) to take. Neither you nor the study team will know which one your child is receiving. They will attend several planned visits to the clinic, where the study team will monitor their health carefully. This will involve regular check-ups, blood tests to measure cholesterol levels and check for safety, and discussions about how your child is feeling and any side effects.
We will explain exactly what's involved at each visit and how long the study will last. There will be continuous support to ensure your child's well-being throughout the study. More detailed information about the schedule of visits and assessments will be provided during the consent process.
Potential risks and benefits
Locations (30)
- Nemours/Alfred I. duPont Hospital for Children ( Site 0001)Verified postcodeWilmington, United States· Recruiting
- Children's National Medical Center ( Site 0015)Verified postcodeWashington D.C., United States· Recruiting
- Excel Medical Clinical Trials ( Site 0008)Verified postcodeBoca Raton, United States· Recruiting
- Children's Healthcare of Atlanta Cardiology ( Site 0026)Verified postcodeAtlanta, United States· Recruiting
- Cincinnati Children's Hospital Medical Center ( Site 0016)Verified postcodeCincinnati, United States· Recruiting
- West Virginia University ( Site 0013)Verified postcodeMorgantown, United States· Recruiting
- Monash Children s Hospital ( Site 1603)Verified postcodeClayton, Australia· Recruiting
- UZ Antwerpen ( Site 0601)Verified postcodeEdegem, Belgium· Recruiting
- Universidade Federal Do Ceara ( Site 0201)Verified postcodeFortaleza, Brazil· Recruiting
- Incor - Instituto do Coracao ( Site 0200)Verified postcodeSão Paulo, Brazil· Recruiting
- Explora Salud ( Site 0304)Verified postcodeSantiago, Chile· Recruiting
- Pontificia Universidad Catolica de Chile ( Site 0300)Verified postcodeSantiago, Chile· Recruiting
Common questions
What is HeFH?
HeFH, or Heterozygous Familial Hypercholesterolemia, is a genetic condition where your body has difficulty removing 'bad' cholesterol (LDL-C), leading to very high levels from childhood.
What is LDL-C?
LDL-C stands for 'low-density lipoprotein cholesterol.' It's often called 'bad' cholesterol because high levels can increase the risk of heart problems.
What is a placebo?
A placebo is a dummy pill that looks exactly like the study medication but contains no active drug. It helps researchers compare the effects of the new medicine.
Will my child get the actual medicine or the placebo?
In this type of study, participants are randomly assigned to either receive the new medicine or the placebo. Neither you nor the study doctors will know which one your child is getting.
Can we stop participating at any time?
Yes, your child's participation is completely voluntary. You can decide to withdraw your child from the study at any point, for any reason, without affecting their medical care.
How to find out more
Toll Free Number
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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