RecruitingOBSERVATIONAL

Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators

This study is called Modulate-CF and it's looking at people with cystic fibrosis (CF) who are taking new medicines called CFTR modulators. These medicines work by helping a protein in the body, called CFTR, do its job better. We want to see how well these medicines are working in people who are already using them as part of their regular care. Researchers will measure specific markers in the body, such as how the gut and nose are working, and the salt levels in sweat. They'll take these measurements before starting the medicine and again after 12 weeks and 52 weeks to understand the long-term effects. This helps us learn more about how these important CF treatments change the body over time.

At a glance

Status

Recruiting

Sponsor

Charite University, Berlin, Germany

Enrolment target

500

Start

01 Jul 2018

Estimated completion

31 Dec 2027

Cystic Fibrosis

What is this study about?

This study, called Modulate-CF, is all about understanding how new treatments for cystic fibrosis (CF) work in the real world. You might have heard of CFTR modulators – these are a type of medicine that helps correct the basic problem in CF by improving how a protein called CFTR functions in your body. This protein is really important for keeping water and salt moving in and out of cells, which stops mucus from becoming too thick.

Researchers want to see exactly how these CFTR modulator medicines, like Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor, and Vanzacaftor-Tezacaftor-Deutivacaftor, affect your body. They'll measure certain 'biomarkers' – these are like internal signs that show how well your CFTR protein is working. By looking at these signs before you start the medicine and then again after you've been taking it for a while, they can understand how much the medicine is helping.

This is an observational study. This means you'll be getting your medicine as part of your normal CF care, and the researchers will simply be observing and collecting information about its effects. They're not giving you a new or experimental drug, but rather studying how the approved ones work in people's everyday lives. This helps doctors and scientists get a better picture of how these important treatments are changing the lives of people with CF.

Key takeaways

This study helps understand how existing CFTR modulator medicines work in real-life settings.
It measures body signs (biomarkers) to see the effects of these medicines.
Participation involves your usual CF care, plus some extra tests at specific times.
Children from 6 months old can take part.
You will be involved for about one year, with tests before starting, then at 12 and 52 weeks.
Your normal CF treatment will not be affected if you choose not to join.

Who may be eligible?

To be part of this study, your doctor and you need to have decided that you will start taking a CFTR modulator medicine as part of your regular cystic fibrosis care. You'll also need to sign a consent form, which explains everything about the study in detail, and if you're a child, you might need to sign an assent form too, showing you understand and want to take part.

You cannot join this study if you are already taking part in another study that is testing a new, unapproved medicine. This is to make sure that the results of this study are clear and only reflect the effects of your CFTR modulator medicine.

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.

Has my doctor and I decided I will start a CFTR modulator medicine?
Am I aged 6 months or older?
Am I willing to sign a consent form (and assent if a child)?
Am I not currently taking part in a study for a new, experimental drug (other than approved CFTR modulators)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will have specific appointments coinciding with your normal CF check-ups. Before you start your CFTR modulator medicine, you'll have some initial tests. These will include measurements of your lung function, looking at your airways with MRI or CT scans, and giving samples of your airway secretions (like mucus). They'll also check your height and weight. Most importantly, specific 'biomarkers' will be measured, which might involve special tests like intestinal current measurement (looking at how your gut is working), nasal potential difference (checking your nose's activity), and a sweat chloride test (measuring salt levels in your sweat).

You will then start your CFTR modulator medicine as advised by your CF doctor. The study team will repeat the biomarker tests at 12 weeks and again at 52 weeks after you start your medication. Other clinical check-ups like lung function, scans, and secretion samples will also be repeated after you've been on the treatment for a specified time. You will continue to take your CFTR modulator medicine throughout the study, as prescribed by your doctor. The total duration of your participation in the study will be about one year.

Potential risks and benefits

The potential benefits of taking part include contributing to a better understanding of how CFTR modulator medicines work in people with cystic fibrosis, which can help improve care for everyone with CF in the future. As you would be receiving treatment that your doctor has already prescribed, this study does not introduce new medical risks from experimental drugs. However, there are minor risks associated with the clinical assessments such as blood tests or imaging, although these are standard procedures. You have the right to withdraw from the study at any time without affecting your normal medical care.

Locations (4)

Charité - Universitätsmedizin Berlin
Verified postcode
Berlin, Germany· Recruiting
Justus-Liebig-University Giessen
Verified postcode
Giessen, Germany· Recruiting
Hannover Medical School
Verified postcode
Hanover, Germany· Recruiting
University of Heidelberg
Verified postcode
Heidelberg, Germany· Recruiting

Common questions

What is a CFTR modulator?

It's a medicine that helps a specific protein in your body called CFTR work better. This protein is faulty in cystic fibrosis.

What are 'biomarkers'?

These are signs in your body, like certain measurements of how your gut or nose works, that help doctors see how treatments affect your condition.

Do I have to take a new medicine if I join?

No, you'll be taking a CFTR modulator medicine that your doctor has already prescribed for you as part of your normal care.

How long will I be in the study?

You'll be involved in the study for about 12 months after starting your CFTR modulator medicine.

Can children join this study?

Yes, children as young as 6 months old can take part if they meet the other requirements.

How to find out more

Simon Y Graeber, MD

simon.graeber@charite.de +4930 450 566 587 Official trial record

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