RecruitingPHASE1INTERVENTIONAL

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

This early-stage clinical trial is investigating a new treatment, VERVE-102, for individuals who have very high cholesterol, either due to a genetic condition called Familial Hypercholesterolemia (HeFH) or because they have developed coronary artery disease at a younger age. The main goal of this first study is to check the safety of VERVE-102 when given as a single dose. VERVE-102 uses advanced gene-editing technology to target a specific gene in the liver, which is hoped to reduce the levels of "bad" cholesterol (LDL-C) in the blood. Researchers will also look at how the body responds to the treatment and if it starts to lower cholesterol levels. This is a crucial step in developing potential new ways to manage very high cholesterol.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Verve Therapeutics, Inc.

Enrolment target

Start

30 Apr 2024

Estimated completion

01 Aug 2027

Heterozygous Familial Hypercholesterolemia Premature Coronary Heart Disease

What is this study about?

This study is exploring a new and innovative treatment known as VERVE-102. It's designed for people who have very high cholesterol that is difficult to manage. Specifically, the study is for those with Heterozygous Familial Hypercholesterolemia (often called HeFH), which is a inherited condition causing extremely high cholesterol from a young age, or those who have developed coronary artery disease (heart problems where blood vessels are narrowed) prematurely.

VERVE-102 works differently from current cholesterol-lowering medications. It uses a cutting-edge technique called 'base-editing' directly in the liver. The idea is to switch off a specific gene called PCSK9. By reducing the activity of this gene, the body should then naturally lower the amount of 'bad' cholesterol (known as LDL-C) circulating in the blood. Managing LDL-C is vital for reducing the risk of heart disease and strokes.

Because this is a very new type of treatment, this first study (Phase 1b) is mainly focused on safety. Researchers will be giving a single dose of VERVE-102 to participants and carefully monitoring them. They will be looking for any side effects and also observing if the treatment starts to show its intended effect on cholesterol levels. This research is a vital step in trying to find new and potentially very effective ways to help people living with challenging high cholesterol conditions.

Key takeaways

Tests a new gene-editing treatment (VERVE-102) for high cholesterol.
Aims to help people with genetic high cholesterol or early heart disease.
Main goal is to check the safety of this new treatment method.
Uses a single dose of the treatment in a first-stage study.
Could offer a new way to lower 'bad' cholesterol (LDL-C).

Who may be eligible?

To be considered for this study, you would need to be an adult between 18 and 70 years old. You must also have either a confirmed diagnosis of Heterozygous Familial Hypercholesterolemia (a genetic condition causing high cholesterol) or have been diagnosed with coronary artery disease (heart conditions) at a younger age.

There are certain reasons why you might not be able to participate. For example, if you have a more severe form of Familial Hypercholesterolemia (called Homozygous), or if you have a history of serious liver problems. Also, if you are currently taking certain types of powerful cholesterol-lowering medications (called PCSK9 inhibitors) or have taken them recently, you might not be eligible.

Finally, the research team will do medical tests, and if any of your results are significantly outside the normal range, as defined by the study guidelines, you may not be able to join. These criteria are put in place to ensure the safety of all participants.

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 18 and 70 years old?
Do you have Familial Hypercholesterolemia (HeFH) OR premature coronary heart disease?
Do you have a history of serious liver problems?
Are you currently taking certain strong cholesterol medications (PCSK9 inhibitors)?
Are your routine blood tests usually within a healthy range?

Answer every question to see your result.

What does participation involve?

If you are eligible and choose to take part in this study, you would receive a single dose of the study drug, VERVE-102. This is an 'open-label' study, which means both you and the study team will know you are receiving the active treatment. You will have a number of visits to the clinic for health checks, blood tests, and scans. These appointments are crucial for monitoring your health, checking for any side effects, and seeing how the treatment is affecting your cholesterol levels. The total duration of your participation, including all follow-up visits, would be explained in full by the study team.

Potential risks and benefits

Participating in any clinical trial involves both potential benefits and risks. There is a potential benefit that VERVE-102 could help to significantly lower your 'bad' cholesterol levels, which could be very impactful for your long-term health. However, because this is a new and investigational treatment, there are also potential risks, including unknown side effects, which the research team will carefully monitor. You will be fully informed of all known and potential risks at every stage, and your safety is the highest priority. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (23)

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

Clinical Study Center
Verified postcode
Dothan, United States· Recruiting
Clinical Study Center
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Pomona, United States· Recruiting
Clinical Study Center
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Boca Raton, United States· Recruiting
Clinical Study Center
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Jacksonville, United States· Recruiting
Clinical Study Center
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Winter Park, United States· Recruiting
Clinical Study Center
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High Point, United States· Recruiting
Clinical Study Center
Verified postcode
DeSoto, United States· Recruiting
Clinical Study Center
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Renton, United States· Recruiting
Clinical Study Center
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Adelaide, Australia· Recruiting
Clinical Study Center
Verified postcode
Melbourne, Australia· Recruiting
Clinical Study Center
Verified postcode
Sydney, Australia· Recruiting
Clinical Study Center
Verified postcode
Chicoutimi, Canada· Recruiting

Common questions

What is Familial Hypercholesterolemia (FH)?

FH is an inherited condition that causes very high levels of 'bad' cholesterol (LDL-C) from birth, leading to a higher risk of early heart disease.

What is 'base-editing' technology?

Base-editing is a very precise gene-editing technique that targets and changes a single 'letter' in our DNA code. In this study, it aims to adjust a specific gene in the liver.

What does a 'single-ascending dose' study mean?

It means participants receive one dose of the treatment. 'Ascending' often refers to different groups receiving slightly higher doses, to find out which dose is safest and most effective.

Will I know if I'm getting the actual treatment?

Yes, this is an 'open-label' study, meaning everyone taking part will receive VERVE-102, and both you and the research team will know this.

Why is lowering 'bad' cholesterol important?

Lowering 'bad' cholesterol (LDL-C) helps to reduce the build-up of fatty deposits in your arteries, which significantly lowers your risk of heart attacks and strokes.

How to find out more

Clinical Development

verve102clinicaltrials@lists.lilly.com 781-970-6833 Official trial record

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