Not yet recruitingOBSERVATIONAL

Heart and Blood Vessels Health in People With Cystic Fibrosis

This study is about understanding how the hearts and blood vessels of people with Cystic Fibrosis (CF) are affected by the condition itself and by new, life-changing CF medicines. CF previously led to a shorter life, but new drugs called 'modulators' mean people are living much longer. This is great news, but doctors are now wondering if heart health might become a bigger concern, especially since these new drugs can sometimes cause weight gain or higher blood pressure, which are known to affect the heart. We will use ultrasound scans to check the health of blood vessels at the start and then again a year later. This helps us see if there are any changes and understand who might be most at risk of heart problems in the future. The aim is to find effective ways to prevent heart disease, which could ultimately improve the health and lifespan of people with CF.

At a glance

Status

Not yet recruiting

Sponsor

Liverpool Heart and Chest Hospital NHS Foundation Trust

Enrolment target

Start

01 Jul 2026

Estimated completion

30 Jun 2028

Cystic Fibrosis (CF)Cardiovascular (CV) Risk Flow-mediated Dilation Evaluation of the Brachial Artery Pulse Wave Analysis

What is this study about?

Cystic fibrosis (CF) is a condition that affects many parts of the body, including the lungs, gut, and other organs. For many years, treatments focused on managing the symptoms. However, new medicines called 'modulators' have made a huge difference, helping people with CF to live much longer and healthier lives. While this is wonderful progress, it also means that doctors are starting to think about other health challenges that might come up as people age with CF.

One area of concern is heart and blood vessel health. Traditionally, heart disease was uncommon in people with CF because of their shorter life expectancy. But with people living longer, and some new CF medicines potentially causing side effects like weight gain or higher blood pressure, there's a need to understand if these factors might increase the risk of heart problems in the CF community. This study aims to look closely at these changes.

We will use special ultrasound scans to check the health of blood vessels in people with CF. These scans are harmless and help us see how well blood flows and if there are any early signs of problems. We'll do these measurements at the start of the study and then repeat them about a year later to see if there are any changes over time. We also want to see if the usual ways doctors predict heart disease risk in the general public are also useful for people with CF. By understanding who might be at higher risk, we can hopefully develop ways to prevent heart problems before they even start, helping people with CF to live even longer and fuller lives.

Key takeaways

This study aims to understand heart and blood vessel health in people with CF.
It will help us see how new CF medicines might affect the heart over time.
We use harmless ultrasound scans and blood tests to gather information.
The goal is to find ways to prevent heart problems in people with CF.
Participation involves two visits, about a year apart, with simple tests.
The study could lead to important discoveries for the long-term health of people with CF.

Who may be eligible?

To be part of this study, you need to have a confirmed diagnosis of cystic fibrosis and be at least 18 years old. You should also be taking certain newer CF medicines (called CFTR modulators, like Elexacaftor/Tezacaftor/Ivacaftor or similar new ones) for at least three months, and be on the correct, prescribed dose. It's important that you are feeling generally well and haven't had a lung infection or been in hospital in the past four weeks.

There's also a group of healthy people needed for comparison. If you are generally well and haven't had a lung infection or been in hospital in the past four weeks, you might be able to take part as a healthy volunteer.

However, some things would mean you couldn't join the study. For example, if you are pregnant or breastfeeding, take long-term steroid medications or certain heart medicines, have severe organ failure, rheumatoid arthritis, or lupus. If you smoke or have smoked in the last 10 years, have had an organ transplant, or have any issues that would make measuring your blood vessels difficult (like an arm injury), you wouldn't be able to participate. You also need to be able to follow instructions for blood tests, which usually involve not eating beforehand.

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 confirmed diagnosis of cystic fibrosis?
Are you currently taking a CFTR modulator medicine (like Kaftrio/Trikafta) and have been for at least 3 months?
Have you been generally well and not had a chest infection or hospital stay in the last 4 weeks?
Are you able to fast (not eat) before a blood test?
Are you male, or if female, are you not pregnant or breastfeeding?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would have two study visits, about a year apart. During each visit, we would use an ultrasound machine to check the health of your blood vessels in your arm. This is a bit like the scans pregnant women have, it's painless and safe. We would also take some blood samples to check various health markers. We'll ask you to answer some questions about your health and lifestyle. You would continue taking your usual CF medicines as prescribed by your doctor. Each visit is expected to take a few hours. The total duration of your involvement would be about one year.

Potential risks and benefits

The main benefit of taking part is helping us understand heart health in people with CF, which could lead to better ways to prevent heart problems in the future. You wouldn't get direct medical treatment through this study, but you might learn more about your own heart health. The risks are generally low; you might feel a small pinch during blood tests and there's a slight chance of bruising, but the ultrasound scans are harmless. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

Liverpool Heart and Chest Hospital
Verified postcode
Liverpool, United Kingdom

Common questions

What is a 'CFTR modulator'?

These are new, effective medicines for cystic fibrosis that help the body's cells work better, improving many CF symptoms.

Will I get any medicine as part of this study?

No, this study is about observing and understanding heart health. You will continue to take your usual prescribed CF medicines.

What does 'flow-mediated dilation' mean?

It's a way of measuring how well your blood vessels can widen. It's a sign of blood vessel health.

How long will each study visit take?

Each visit is expected to take a few hours to complete all the tests.

Can I stop participating if I change my mind?

Yes, you can choose to leave the study at any time without needing to give a reason, and it won't affect your medical care.

How to find out more

Alex Chan

alex.chan@lhch.nhs.uk +4401516001416 Official trial record

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