Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of MK-0616 in Adults With Heterozygous Familial Hypercholesterolemia

This research study is looking at a new medicine called MK-0616, given as a tablet, for adults who have a genetic condition called Heterozygous Familial Hypercholesterolemia (HeFH). This condition causes very high levels of 'bad' cholesterol in the blood. The main goals are to find out how well MK-0616 lowers 'bad' cholesterol (LDL-C) after 24 weeks and to check for any side effects. To do this, some people will receive the new tablet, and others will receive a dummy tablet (placebo), without knowing which they are getting. We will compare these two groups to understand if MK-0616 is effective and safe. This is a common way to test new medicines.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Merck Sharp & Dohme LLC

Enrolment target

123

Start

25 Sep 2023

Heterozygous Familial Hypercholesterolemia (HeFH)

What is this study about?

This study is about a new medicine called MK-0616, which is given in tablet form. It's for adults who have a specific health condition known as Heterozygous Familial Hypercholesterolemia, often shortened to HeFH. This is a genetic condition, meaning it's passed down in families, and it causes people to have very high levels of cholesterol in their blood, especially a type called 'LDL-C' or 'bad' cholesterol. High levels of 'bad' cholesterol can increase the risk of heart problems.

The main aim of this study is to see if MK-0616 can help lower this 'bad' cholesterol effectively and safely. To do this fairly, we'll compare it to a 'placebo', which is a dummy tablet that looks the same but contains no medicine. Neither you nor your study doctor will know if you're taking the actual medicine or the placebo. This is called a 'double-blind' study and helps us get the most accurate results by preventing bias.

Researchers will closely watch how much your 'bad' cholesterol changes after 24 weeks on the treatment. They will also keep a close eye on any side effects you might experience. This means they will record any new health problems or discomfort you have during the study. This information is really important to understand if MK-0616 could be a good new medicine for people with HeFH.

Key takeaways

This study is testing a new tablet for a genetic high cholesterol condition (HeFH).
It aims to see how well the medicine lowers 'bad' cholesterol and if it's safe.
Participants will receive either the new medicine or a dummy tablet.
Regular clinic visits and blood tests will be part of the study.
It includes adults aged 18 and over, both men and women, with HeFH.

Who may be eligible?

To join this study, you must be an adult, aged 18 years or older. There is no upper age limit, so people of all adult ages can be considered. Both men and women are welcome to take part in this research.

All participants will need to have a confirmed diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH). This is a specific type of high cholesterol that runs in families. The study team will check your medical history and conduct some tests to make sure you meet all the necessary health requirements for the study. They will also ensure that it's safe for you to take part.

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?
Do you have a diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)?
Are you able to take tablets regularly as prescribed?
Are you able to attend regular clinic appointments for check-ups and tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be randomly assigned to one of two groups: one group will receive the new medicine, MK-0616, in tablet form, and the other group will receive a dummy tablet (placebo). You won't know which one you're getting, and neither will your study doctor. You'll need to take the tablets as instructed.

Throughout the study, you'll have regular visits to the clinic. At these visits, the study team will take blood samples to measure your cholesterol levels, check your general health, and ask about any side effects you might be experiencing. These assessments are important to see if the medicine is working and if it's safe. The study will last for a period of time, with cholesterol levels checked at various points, particularly at 24 weeks and then again at 52 weeks to see the longer-term effects. There will be follow-up appointments after you stop taking the study medication to ensure your well-being.

Potential risks and benefits

Taking part in a study like this means you might benefit from a new treatment for your condition before it's widely available. You'll also receive careful monitoring of your health from the study team. However, there's no guarantee that the new medicine will work for you, or that you'll even receive the active medicine. Like all medicines, MK-0616 might have side effects, and some could be unpleasant or serious. All risks and potential benefits will be explained in detail by the study team. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

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

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Unverified
Finland
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Unverified
Norway
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Unverified
Spain
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Unverified
Netherlands
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Unverified
Czechia
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Unverified
Hungary

Common questions

What is HeFH?

HeFH stands for Heterozygous Familial Hypercholesterolemia. It's a genetic condition that causes very high levels of 'bad' cholesterol (LDL-C) in your blood from birth, making heart problems more likely.

What is 'bad' cholesterol (LDL-C)?

LDL-C is often called 'bad' cholesterol because high levels can lead to a build-up in your arteries, increasing your risk of heart disease.

What is a 'placebo'?

A placebo is a dummy pill that looks just like the real medicine but contains no active drug. It helps researchers compare the effects of the actual medicine fairly.

Will I know if I'm getting the real medicine?

No, neither you nor your study doctor will know if you're taking the new medicine or the placebo. This is a standard way to get unbiased results.

What happens if I have side effects?

The study team will monitor you closely for any side effects. You should report any new or worsening symptoms, and they will provide care and guidance.