RecruitingPHASE1INTERVENTIONAL

Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

This study is looking into a new treatment called VERVE-201 for adults aged 18 to 70 who have extremely high levels of cholesterol and other fats (triglycerides) in their blood that haven't improved with other treatments. It's a 'Phase 1b' trial, meaning the main goal is to check how safe VERVE-201 is and how well the body tolerates it when given in different doses. VERVE-201 uses a clever technology to naturally lower these blood fats by switching off a specific gene in the liver. The study will monitor patients closely to understand how this new approach works in the body and if it can effectively reduce harmful cholesterol and triglyceride levels.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Verve Therapeutics, Inc.

Enrolment target

Start

30 Oct 2024

Estimated completion

01 Dec 2027

Hypercholesterolemia Hypertriglyceridemia Refractory Hyperlipidemia

What is this study about?

This study is a first-step clinical trial investigating a new potential medicine called VERVE-201. It's for people who have seriously high levels of cholesterol and other fats in their blood – a condition often called refractory hyperlipidemia. This means that despite trying other available treatments, their levels remain stubbornly high. High cholesterol and fats can increase the risk of heart problems, so finding new ways to manage them is really important.

VERVE-201 is quite innovative because it uses a technology called 'base editing'. Think of it like a very precise genetic 'spell checker' that can make a tiny change to a specific gene in the liver called ANGPTL3. The aim is to switch off this gene, which then helps the body to naturally lower levels of LDL cholesterol (often called 'bad' cholesterol) and triglycerides (another type of fat) in the blood. This study is designed as an 'open-label, single-ascending dose' trial. This means both you and your doctor will know you are receiving VERVE-201, and researchers will give different groups of patients slightly different doses to find the safest and most effective amount.

The main goal of this initial study is to check the safety of VERVE-201. Researchers will be carefully watching for any side effects and how well people tolerate the treatment. They also want to understand how the medicine works in the body to lower cholesterol and triglyceride levels. This early research is crucial to determine if VERVE-201 is a promising option that could eventually help many people manage their high blood fat levels more effectively.

Key takeaways

New study for very high cholesterol/fats not responding to other treatments.
Investigates VERVE-201, a new gene-editing therapy.
Aims to safely lower 'bad' cholesterol and triglycerides.
Primary focus is on safety and how the treatment works in the body.
Open to adults aged 18-70 with specific very high blood fat conditions.
Participation involves close monitoring after a single treatment dose.

Who may be eligible?

To be considered for this study, you would need to be an adult between 18 and 70 years old. Most importantly, you must have very high cholesterol and/or very high triglycerides that haven't responded well to standard treatments – this is what "refractory hyperlipidemia" means in simple terms. Both men and women can take part.

There are also some reasons why you might not be able to join. For example, if you have a current or past history of serious liver disease, or if you're currently receiving or have recently received a specific type of antibody treatment that targets the ANGPTL3 gene. Researchers will also check your blood test results and overall health to make sure there are no other significant medical issues that would make it unsafe for you to participate.

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 very high cholesterol or triglycerides that haven't responded to previous treatments?
Have you ever had serious liver disease?
Are you currently or have you recently received a specific antibody treatment for ANGPTL3?
Are you able to attend regular follow-up appointments?

Answer every question to see your result.

What does participation involve?

If you are eligible and choose to take part, you will receive VERVE-201 as a single dose. Researchers will closely monitor your health before, during, and after you receive the medication. This will involve regular clinic visits, blood tests, and other health checks to see how you are responding and to make sure the treatment is safe. The full duration of your participation, including all follow-up appointments, will be explained in detail by the study team, but typically, these early-stage studies involve careful monitoring over several months.

Potential risks and benefits

Participating in a clinical trial might offer potential benefits, such as gaining early access to a new treatment that could help manage your high blood fats when other options haven't worked. However, like all new medicines, there are potential risks, including side effects that are not yet fully known. The study team will explain all known risks to you in detail. You are always free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (10)

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

Clinical Study Center
Verified postcode
Adelaide, Australia· Recruiting
Clinical Study Center
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Melbourne, Australia· Recruiting
Clinical Study Center
Verified postcode
Chicoutimi, Canada· Recruiting
Clinical Study Center
Verified postcode
Montreal, Canada· Recruiting
Clinical Study Center
Verified postcode
Toronto, Canada· Recruiting
Clinical Study Center
Verified postcode
Cape Town, South Africa· Recruiting
Clinical Study Center
Verified postcode
Johannesburg, South Africa· Recruiting
Clinical Study Center
Verified postcode
Liverpool, United Kingdom· Recruiting
Clinical Study Center
Verified postcode
London, United Kingdom· Recruiting
Clinical Study Center
Verified postcode
Manchester, United Kingdom· Recruiting

Common questions

What does 'refractory hyperlipidemia' mean?

It means you have very high levels of cholesterol and other fats in your blood that haven't gone down enough with other treatments you've tried.

What is VERVE-201 and how does it work?

VERVE-201 is a new experimental medicine. It uses a technology to 'switch off' a specific gene in your liver, which scientists hope will naturally lower your 'bad' cholesterol and other blood fats.

Is this a new type of treatment?

Yes, it uses a very advanced genetic editing technology. This is one of the very first studies to test it in people, so it's quite new.

What is the main purpose of this study?

The main purpose is to check how safe VERVE-201 is for patients and to see how the body reacts to it, especially how it affects cholesterol and fat levels.

Can I take part if I also have liver problems?

Unfortunately, no. If you have a current or past history of serious liver disease, you would not be able to participate for safety reasons.

How to find out more

Clinical Development

verve201clinicaltrials@lists.lilly.com 781-970-6833 Official trial record

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