Active not recruitingPHASE4INTERVENTIONAL

Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)

The RECOVER study is watching how a new medicine called Kaftrio helps people with cystic fibrosis (CF) in their daily lives. It's not a standard drug trial; instead, it's observing people already taking Kaftrio. Over two years, researchers will check how the medicine affects things like lung health, tummy issues, and general well-being. They'll also look at how often people take their medicine and if it improves their quality of life. The study plans to include children aged 6 and up, but will start with those aged 12 and older who are already prescribed Kaftrio by their doctor. The main goal is to understand the real-world benefits of this important treatment.

At a glance

Status

Active not recruiting

Phase

PHASE4

Sponsor

Royal College of Surgeons, Ireland

Enrolment target

237

Start

03 Sep 2020

Estimated completion

01 Jul 2029

Cystic Fibrosis Cystic Fibrosis Liver Disease Cystic Fibrosis in Children Adherence, Medication Cystic Fibrosis Gastrointestinal Disease

What is this study about?

The RECOVER study is an important project looking into a medicine called Kaftrio, which is used to treat cystic fibrosis (CF). Instead of testing if the medicine works, like in a new drug trial, this study is observing people who are already taking Kaftrio in their everyday lives. It's called a 'real-world' study because it aims to see how the medicine performs for patients and their families outside of very controlled research settings.

Over two years, the study will carefully track many aspects of health for people with CF who are taking Kaftrio. This includes checking how well their lungs are working, looking at tummy problems, and understanding their overall health and quality of life. They will also collect samples like blood, spit, and stool to get a deeper understanding of how Kaftrio changes the body. The study starts with people aged 12 and over, and will include children aged 6 and up once Kaftrio is approved for their age group.

The main goal of RECOVER is to gain a really detailed picture of how Kaftrio helps people with CF. By looking at many different health measures, including some that aren't usually checked in standard trials, the researchers hope to learn exactly how Kaftrio improves symptoms and the lives of those living with cystic fibrosis. This information will be very valuable for doctors and patients in the future.

Key takeaways

RECOVER studies how Kaftrio works for people with CF in everyday life.
It lasts for two years and involves regular health checks.
You must already be prescribed Kaftrio to be eligible.
The study includes those aged 12+ now, and 6+ later.
Information gathered will help doctors understand Kaftrio's benefits.

Who may be eligible?

To be part of the RECOVER study, you would need to be someone living with cystic fibrosis (CF).

Initially, the study is inviting people aged 12 and older who have been prescribed Kaftrio by their doctor. Your doctor must have decided that Kaftrio is the right medicine for you, following the usual guidelines. For children aged 6 to 11, they can join the study later, but only when Kaftrio has been officially approved and funded for their age group. Similarly, they must be taking Kaftrio as prescribed by their doctor.

It's important that anyone joining the study is taking the full, recommended dose of Kaftrio for their age. If you've just started taking Kaftrio, or even if you've been on it for a while and your doctor has collected some health information already, you might still be able to join. Your doctor can discuss if this study is suitable for you.

Quick self-check

I have cystic fibrosis (CF).
I am currently taking Kaftrio.
My doctor has prescribed Kaftrio for me.
I am taking the full recommended dose of Kaftrio.
I am 12 years old or older (or 6-11 if Kaftrio is approved for my age group).

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in RECOVER, you'll be followed for two years. During this time, you'll attend regular appointments where different assessments will be done. These assessments include simple things like measuring your height, weight, and how well you can breathe (lung function tests). You might also have scans of your lungs or tummy, and give samples like spit, stool, or nasal wash. You'll complete questionnaires about your symptoms and how you're feeling overall.

The study focuses on people who are already taking Kaftrio as prescribed by their own doctor. This means you won't be given Kaftrio as part of the study; your doctor will still manage your medication as normal. The study is simply observing the effects of the treatment you're already receiving. The doctors leading the study, Paul McNally and Jane Davies, along with their teams, will collect all this information to understand how Kaftrio is working for you and others.

Potential risks and benefits

Taking part in this study means doctors will closely monitor your health while you’re on Kaftrio, which could give you a better understanding of your own progress. The information gathered will also help improve care for all people with CF in the future. As you're already taking Kaftrio as part of your standard care, the study itself doesn't introduce new medical risks from the drug. However, some tests, like providing samples or having scans, might cause slight discomfort or take up some of your time. You are always free to change your mind and withdraw from the study at any point, without affecting your regular medical care.

Locations (7)

Children's Health Ireland at Crumlin
Dublin, Ireland
Children's Health Ireland at Temple Street
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
University Hospital Limerick
Limerick, Ireland
Children's Health Ireland at Tallaght
Tallaght, Ireland
Royal Belfast Hospital for Sick Children
Belfast, United Kingdom
Royal Brompton Hospital
London, United Kingdom

Common questions

What is the purpose of the RECOVER study?

It's to see how well the medicine Kaftrio works in real-life for people with cystic fibrosis (CF).

Who can join the study?

Initially, people with CF aged 12 and over who are already taking Kaftrio, with plans to include children aged 6-11 later.

Will I get Kaftrio through this study?

No, you must already be prescribed Kaftrio by your own doctor before joining the study.

How long will the study last if I join?

You will be followed by the study for two years.

What kind of tests will I have?

You might have breathing tests, scans, give samples like spit or stool, and fill out questionnaires.