CompletedPhase IVInterventional

Can people with cystic fibrosis safely stop taking some of their nebulised treatments if they are established on the new modulator therapy, Kaftrio?

Many people with cystic fibrosis spend hours each day inhaling nebulised medicines to clear thick mucus from their lungs. The newer combination medicine, Kaftrio, has significantly improved lung health for many patients. Because Kaftrio works so well, patients and doctors now want to know if it is safe to reduce other daily treatments. The CF STORM study aims to answer this question. It involves people aged six and older who have been taking Kaftrio for at least three months. Half of the participants will carry on taking their regular mucus-clearing nebulisers, while the other half will stop them. Over 12 months, researchers will check lung function through standard breathing tests during regular clinic visits or at home. The results will help people with cystic fibrosis and their care teams make safe, informed choices about reducing daily treatment burdens.

At a glance

Status

Completed

Phase

Phase IV

Sponsor

Alder Hey Children's NHS Foundation Trust

Enrolment target

764

Start

21 Jul 2021

Estimated completion

30 May 2024

Cystic fibrosis

What is this study about?

Living with cystic fibrosis usually means spending a lot of time on daily treatments. For many years, people have relied on nebulisers to inhale medicines like dornase alfa or hypertonic saline. These treatments help break down thick, sticky mucus in the lungs to prevent chest infections. While effective, setting up, taking, and cleaning nebulisers every day can feel exhausting and take away hours from work, school, and family life.

Recently, modern medicines called CFTR modulators—particularly Kaftrio—have transformed cystic fibrosis care. Rather than just clearing mucus after it builds up, Kaftrio helps treat the underlying cause of cystic fibrosis. Because Kaftrio has improved lung health so significantly, both patients and doctors have started asking an important question: is it still necessary to keep taking all of those daily nebulisers?

The CF STORM trial was created to find out. Led by UK doctors and researchers, this trial looks closely at what happens to lung function when people established on Kaftrio stop their daily mucus-thinning nebulisers. By gathering clear evidence across the UK, the study aims to give patients confidence about which treatments they truly need, potentially freeing up valuable time each day.

Key takeaways

Tests whether people on Kaftrio can safely stop time-consuming mucus-thinning nebulisers.
Open to people with cystic fibrosis in the UK aged six years and older.
Lasts for 12 months and fits into your normal clinic schedule with no extra hospital visits required.
Half of the participants stop their nebulisers while the other half continue them.
Patients are monitored closely, and nebulised treatments can be restarted at any time if needed.

Who may be eligible?

This trial is open to adults and children aged six and older who have cystic fibrosis and have been taking Kaftrio for at least three months. Participants must also have been using daily mucus-clearing nebulisers (such as dornase alfa, hypertonic saline, or both) for at least three months. You need to be registered on the UK CF Registry and able to perform standard breathing tests (spirometry). Your chest should also be reasonably stable, meaning you have not needed extra antibiotics in the two weeks before joining.

You cannot take part if you have had a severe reaction to Kaftrio that needed a dose change in the last three months, or if your lung function has dropped below 40% during that time. The trial is also not suitable for people who have had an organ transplant, are currently on an active transplant waiting list, take mannitol dry powder inhalers, or have other serious medical conditions that could interfere with their care.

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.

I am aged 6 years or older and diagnosed with cystic fibrosis.
I have been taking Kaftrio for at least 3 months.
I take daily mucus-clearing nebulisers (like dornase alfa or hypertonic saline).
I am signed up to the UK CF Registry and can do spirometry breathing tests.
I have not needed extra antibiotics in the past 2 weeks.
My lung function (FEV1) is at or above 40% and I have not had an organ transplant.

Answer every question to see your result.

What does participation involve?

Taking part lasts for 12 months. When you join, a computer randomly places you into one of two groups: one group continues their usual mucus-thinning nebulisers, while the other group stops them. Both groups carry on taking Kaftrio as normal.

The trial is designed to fit around your ordinary NHS care, meaning you will not have to make extra hospital trips. Your breathing tests, health reviews, and routine check-ups will happen during your normal clinic visits or can even be done remotely from home. You will also be asked to complete brief questionnaires about your quality of life. If your nebuliser records your use electronically, that information may be shared securely with the research team. Your cystic fibrosis team will monitor your lungs carefully throughout the entire year.

Potential risks and benefits

Taking part may not directly benefit your health, but it could help prove whether people on Kaftrio can safely stop time-consuming nebulisers, helping you and others reduce treatment burdens in the future. The main risk is that your chest symptoms could flare up or your lung function could dip after stopping your nebulisers. To protect your safety, your health will be tracked closely, and your doctor can restart your nebulisers at any time if necessary. Taking part is entirely voluntary, and you can change your mind and leave the study at any point without it affecting your standard NHS care.

Locations (6)

Alder Hey Hospital
Approximate
Liverpool, England
John Radcliffe Hospital
Approximate
Oxford, England
Great Ormond Street Hospital
Approximate
London, England
Liverpool Heart And Chest Hospital
City only
Liverpool, England
Manchester University NHS Foundation Trust
City only
Manchester, England
Belfast City Hospital
Unverified
Belfast, Northern Ireland

Common questions

Will I have to stop taking Kaftrio during the trial?

No. Everyone in the study continues to take their Kaftrio as prescribed. The trial only tests whether you can stop taking additional mucus-thinning nebulisers.

Can I choose which group I am in?

No. A computer allocates participants randomly to either continue or stop their nebulisers. This ensures the results are fair, accurate, and scientifically reliable.

Will I need to visit the hospital more often?

No. The study is planned around your normal clinic appointments, and many parts of the study—including consent and check-ins—can be done remotely from home.

What happens if my chest feels worse during the trial?

Your safety comes first. If your symptoms worsen or your breathing tests drop, your cystic fibrosis team will review your health and can restart your nebulisers straight away.

Does it cost anything to take part?

No. The trial is part of NHS care and is funded by the National Institute for Health and Care Research (NIHR), so there are no costs to you.

How to find out more

Abigail Williams

cfstorm@liverpool.ac.uk +44 151 794 9764 Official trial record

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