Active not recruitingOBSERVATIONAL

Assessment of CFTR-Modulator Treatment in Cystic Fibrosis Lung Disease Using Novel Structural and Functional MRI

This research study aims to understand how a special type of scan, called an MRI, can show changes in the lungs of people with cystic fibrosis (CF). Researchers want to see if MRI can detect improvements or changes when people with CF start new treatments called CFTR-modulators. The study includes children and young people aged 4 to 18, both those with CF and healthy volunteers. It involves several visits over time, where participants will have lung tests, MRI scans, and answer questions about their quality of life. The main goal is to see if MRI can be a good way to track how well new CF treatments are working.

At a glance

Status

Active not recruiting

Sponsor

The Hospital for Sick Children

Enrolment target

Start

20 Oct 2020

Estimated completion

30 Jun 2026

Group 1: Healthy Controls Group 2: Stable CF Group 3: CF Participants Anticipated to Receive CFTR-modulator Therapy Group 4: 4-8 yo CF Participants Starting Triple Combination Modulator Therapy

What is this study about?

This study is being carried out to see if a special type of scan, called an MRI (Magnetic Resonance Imaging), can help us better understand cystic fibrosis (CF) and how new treatments affect the lungs. CF is a condition that mainly affects the lungs and digestive system, making mucus thick and sticky. New medicines, called CFTR-modulators, are available that can help improve how the lungs work for many people with CF.

The researchers are particularly interested in seeing if MRI can pick up changes in the structure and function of the lungs in people when they start these new CFTR-modulator medicines. This is important because if MRI proves to be a good way to measure these changes, it could help doctors and researchers in the future to better monitor how well treatments are working and when to adjust them. It might also help speed up the development of even better treatments.

The study involves different groups of people: healthy volunteers, people with CF who are stable, and people with CF who are about to start, or have recently started, CFTR-modulator therapy, including children aged 4-8. By comparing these different groups, the researchers hope to get a clear picture of how CF affects the lungs and how new treatments make a difference. This information could ultimately improve care for people with CF.

Key takeaways

This study uses MRI to understand how CFTR-modulator treatments affect CF lungs.
It aims to improve future monitoring and treatment for people with CF.
Participation involves multiple visits for breathing tests, MRI, and quality-of-life questionnaires.
It's an observational study, meaning no new medicines are given as part of the research.
Open to individuals with CF and healthy controls, aged 4 to 18.
You can withdraw from the study at any time without consequences.

Who may be eligible?

To join this study, you or your child must be at least 4 years old. If you're under 18, a parent or guardian will need to give permission, and the child will also need to agree to take part if they're old enough to understand.

You also need to be able to do breathing tests where you blow into a machine, and be able to hold your breath for short periods during the MRI scan. It's important that you don't have certain medical conditions that would make it unsafe to do the tests, and your lung function (measured by a test called FEV1) should not be extremely low – it needs to be 40% or more of what's expected for your age.

There are also a few reasons why you might not be able to join. For example, if you get very anxious in small spaces (claustrophobia), or if you don't meet the safety requirements for an MRI scan. You shouldn't have had a cough in the 3 days before a study visit, or taken oral antibiotics in the 3 weeks before a visit, as these could affect the test results.

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 or your child aged 4 years or older?
Can you/your child comfortably hold your breath for short periods?
Are you/your child generally stable and don't have any serious medical issues that would prevent you from doing the tests?
Do you/your child have an FEV1 (a lung function measure) of 40% or higher?
Are you/your child comfortable with enclosed spaces and don't suffer from severe claustrophobia?
Have you/your child avoided coughing in the last 3 days and oral antibiotics in the last 3 weeks?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll have several visits to The Hospital for Sick Children (SickKids) in Canada. For most participants, there will be 3 visits initially: one at the start (baseline), one about a month later, and another 6 months after that. If you're happy to continue, there's a chance to extend for up to three more visits at 1, 2, and 3 years from the start.

At each visit, you'll have a few different tests. This includes breathing tests (spirometry and sometimes body plethysmography), a special test called multiple breath washout, and the main part of the study: an MRI scan of your lungs. You'll also be asked to fill out questionnaires about how you're feeling and your quality of life. The study design doesn't involve giving you any new medication; it's focused on observing changes when you're already on a CFTR-modulator or if you're a control participant.

Potential risks and benefits

Taking part in this study could offer some benefits, such as contributing to medical knowledge that may improve the care of people with cystic fibrosis in the future. You'll also receive regular monitoring of your lung health. Potential risks are generally low, mainly related to the minor discomforts of breathing tests or the MRI scan, such as lying still for a period. There are no known risks from the MRI itself, as it doesn't use X-rays. You'll be asked to answer questions about your well-being, and some people might find this a little personal. Remember, your participation is voluntary, and you can choose to withdraw from the study at any time without affecting your usual medical care.

Locations (1)

The Hospital for Sick Children
Verified postcode
Toronto, Canada

Common questions

What is an MRI and is it safe?

An MRI (Magnetic Resonance Imaging) uses strong magnets and radio waves to create detailed pictures inside your body. It does not use X-rays or radiation and is generally considered very safe.

Will I get medication in this study?

No, this study is about observing the effects of treatments you might already be taking or are about to start. It does not provide new medications.

How long will each study visit take?

The study brief summary doesn't specify the exact length of each visit, but they will include several tests like breathing tests and an MRI scan, so they are likely to take a few hours.

If I have CF, will my medical care change if I join?

No, participating in this study will not change the medical care you receive from your usual doctors. You can continue with your regular treatment plan.

What if I feel uncomfortable during the MRI scan?

The study team will be there to support you. If you feel uncomfortable, you can communicate with them. If you have severe claustrophobia, you might not be suitable for the study.