CompletedPhase IVInterventional

Real world health outcomes in people with cystic fibrosis after initiation of a new combination treatment

Cystic fibrosis causes thick, sticky mucus to build up in the lungs and digestive system. In recent years, powerful new combination medicines have been developed that treat the root cause of the condition. This long-term study, called RECOVER, is looking at how these treatments work in everyday life outside of strict clinical trials. Researchers are tracking participants aged six and older across specialist centres in the UK and Ireland over seven years. Your own doctor will decide whether these medicines are right for you as part of your normal NHS or routine care. If you choose to join, researchers will monitor your health using regular clinic visits, breathing checks, and occasional scans. The results will help doctors better understand the long-term benefits of these treatments for people with cystic fibrosis.

At a glance

Status

Completed

Phase

Phase IV

Sponsor

Royal College of Surgeons in Ireland

Enrolment target

254

Start

01 Aug 2020

Estimated completion

31 Jul 2023

Cystic fibrosis

What is this study about?

Cystic fibrosis is an inherited condition that affects the way salt and water move in and out of cells. This causes thick, sticky mucus to collect in the lungs and gut, leading to chest infections and problems digesting food. Recently, a new group of medicines known as combination modulators has become available. These medicines target the faulty protein that causes cystic fibrosis, helping cells work much more normally.

While early clinical trials showed these medicines work very well, those studies only followed a small group of carefully selected patients for a short time. This study, known as RECOVER, is a 'real-world' study. That means it looks at how the medicines work in everyday life across a much wider group of people, including those with other health conditions or different levels of lung health.

Over seven years, researchers will gather vital information about how these treatments affect lung function, infection rates, and general quality of life. The knowledge gained from this study will help healthcare teams provide better, more tailored care for children and adults living with cystic fibrosis in the years to come.

Key takeaways

The RECOVER study tracks how new cystic fibrosis medicines work in daily life over seven years.
Open to adults and children aged six and older across the UK and Ireland.
Your regular doctor decides your medication as part of your normal care.
Involves check-ups every three months for two years, then once a year.
Participation is entirely voluntary and you can withdraw at any time.

Who may be eligible?

You may be able to take part if you are aged six or older and have a diagnosis of cystic fibrosis. Your regular doctor must have already decided that a triple-combination medicine is right for you, based on normal medical guidelines. You might be starting one of these medicines for the first time or switching to a newer version.

Because this is a real-world study, the rules for joining are quite open. You can take part even if your lung function is very high or quite low, or if you have other medical conditions or long-standing chest infections that might have kept you out of earlier trials.

If you are of childbearing age, you may need to use reliable contraception depending on local guidelines and standard safety rules. A parent or guardian will need to give permission for children to take part, alongside the child's own agreement.

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 have been diagnosed with cystic fibrosis.
I am aged six or older.
My doctor has already recommended and prescribed a triple-combination medicine for me.
I am willing to attend clinic visits and complete breathing checks over several years.
I am happy to follow local guidance on contraception if this applies to me.

Answer every question to see your result.

What does participation involve?

Taking part in this study lasts for up to seven years, but it fits closely alongside your usual hospital care. Your medication is prescribed by your own doctor through normal healthcare routes, not by the study team itself.

At the start of the study, you will have a baseline check-up. For the first two years, you will attend study visits every three months, often timed with your regular clinic appointments. After two years, visits will usually happen just once a year.

During visits, the team will check your health and may ask you to do some tests. These can include breathing tests, giving a sputum (phlegm) sample, and a gentle salt-water nose wash. Blood tests will be done, but the team will try to collect these only when you are already having blood taken for your routine care. You may also have chest CT scans so doctors can take detailed pictures of your lungs.

Potential risks and benefits

Taking part does not guarantee any personal health improvements, but you will be monitored very closely by your cystic fibrosis team, which can be reassuring and helpful for your care. The study tests carry minor discomforts: the nose wash and phlegm tests can cause mild irritation or coughing, and blood tests may cause brief stinging or bruising. The study CT scans use low doses of radiation, which very slightly increases your natural lifetime risk of cancer (from about 50% to 50.1%). Taking part is completely voluntary, and you can stop at any time without your regular NHS care or medication being affected.

Locations (2)

Guy's and St Thomas' NHS Foundation Trust
City only
London, England
Royal Belfast Hospital for Sick Children
Approximate
Belfast, Northern Ireland

Common questions

Do I have to pay for the study medicines?

No. Your treatment is prescribed through your regular doctor and provided through your normal healthcare service, such as the NHS.

Will taking part change my normal treatment plan?

No. Your cystic fibrosis doctor remains in charge of your care, and any medical decisions are made independently of the research study.

What happens if I want to leave the study?

You are free to leave the study at any point. Leaving will not change your relationship with your healthcare team or your access to treatment.

Are the extra CT scans safe?

CT scans use a small amount of radiation. The extra risk to your health is very small, but your doctor will discuss this with you before you agree to take part.

Can my child join if they are under 18?

Yes, children aged six and older can join if their doctor decides the treatment is right for them. Parents must give consent, and the child will also be asked for their agreement.

How to find out more

Rachel Cregan

rachelcregan@rcsi.ie +353 (0)86 195 3073 Official trial record

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