RecruitingOBSERVATIONAL

Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following Newborn Screening

This study is investigating how lung disease caused by cystic fibrosis (CF) starts and develops in babies and toddlers. Specifically, it aims to understand the early stages of CF lung problems in children who are diagnosed with CF soon after birth following newborn screening. The study will also compare these children with those diagnosed later in life due to symptoms, to see if early diagnosis and treatment make a difference. Researchers will collect information from routine check-ups like lung function tests and scans, along with regular phone interviews about breathing problems. The goal is to learn more about how CF affects the lungs from a very young age, which could help improve care in the future.

At a glance

Status

Recruiting

Sponsor

Heidelberg University

Enrolment target

200

Start

01 Dec 2011

Estimated completion

01 Dec 2030

Cystic Fibrosis Lung Disease

What is this study about?

Cystic fibrosis (CF) is a serious genetic condition that affects many parts of the body, especially the lungs. For many people with CF, lung problems are the main reason for health difficulties later in life. Although treatments have improved and people with CF are living longer, scientists still don't fully understand how lung disease starts and progresses in very young children with CF.

This study, called a 'longitudinal observational study', aims to change that. Researchers want to closely watch and learn about how CF lung disease develops in babies and toddlers. They are particularly interested in children who are diagnosed with CF very early, often through newborn screening tests. By understanding these early stages better, they hope to find ways to improve care and treatments from a young age.

The study will collect information from children who have CF, including details from their regular check-ups, such as lung scans and breathing tests. They will also speak to parents regularly about their child's symptoms and health. By looking at all this information over time, the researchers hope to gain a much clearer picture of what happens to the lungs in the first few years of life with CF. This knowledge is really important for developing better ways to help children with CF live healthier lives.

Key takeaways

This study aims to understand how CF lung disease starts in babies and young children.
It uses information from regular check-ups and extra phone calls/questionnaires.
Compares children diagnosed early versus those diagnosed later with CF.
Mainly involves observing health over time, not many new procedures.
The goal is to improve future care and treatments for children with CF.

Who may be eligible?

This study is looking for children who have recently been diagnosed with cystic fibrosis (CF). To be included, their CF diagnosis must be clearly confirmed through specific medical tests, like a sweat test or genetic test.

Children can be included if they were diagnosed early in life, either through newborn screening or for other reasons like family history, before they were 4 months old (or corrected age if they were born prematurely). Children diagnosed with CF after 4 months of age due to symptoms can also join.

However, some children might not be suitable for the study. This includes very premature babies, those who needed long-term breathing support in their first few months, or children with other major health conditions that might interfere with the study. Also, if a child or their parents don't want to take part, or if the CF diagnosis isn’t certain, they wouldn't be able to join.

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 your child recently been diagnosed with Cystic Fibrosis (CF)?
Is your child's CF diagnosis confirmed by a sweat test, genetic test, or other specific medical findings?
Was your child either diagnosed before 4 months of age (or corrected age for premature babies), OR diagnosed after 4 months old due to symptoms?
Does your child NOT have other major health issues that aren't related to CF, especially other long-term lung conditions?
Are you and your child willing to regularly share information about their health with the study team?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will have their health information collected regularly over time. This will mainly involve using data from their usual annual check-ups at the hospital. These check-ups typically include things like chest scans (like an MRI), tests to see how well their lungs are working, and samples for microbiology (to check for germs). They will also measure their height, weight, and other general health markers.

In addition to these routine appointments, a study nurse will contact you by phone once a month to ask questions about your child's breathing and any symptoms they might have. Every three months, you will also be asked to complete a questionnaire about your child's overall health and quality of life. Sometimes, doctors might suggest an optional procedure called a bronchoscopy with lavage (where a small tube is used to look at the airways and collect samples) as part of their care, and if this happens, the samples could also be used for the study. The study will continue for an extended period, gathering information as your child grows.

Potential risks and benefits

Taking part in this study may offer a better understanding of your child's own health journey with CF, as information from their regular care will be carefully reviewed. There aren't many extra procedures specifically for the study, beyond phone calls and questionnaires, so the main risks would be related to any routine clinical assessments that are happening anyway. The knowledge gained from this study is expected to help doctors understand more about how CF affects children's lungs from a young age, potentially leading to better care for all children with CF in the future. Remember, participating is always your choice, and you are free to withdraw your child from the study at any time without affecting their medical care.

Locations (4)

University Children's Hospital Heidelberg, Cystic Fibrosis Centre
Verified postcode
Heidelberg, Germany· Recruiting
University Hospital Gießen and Marburg GmbH
Verified postcode
Giessen, Germany· Recruiting
Medizinische Hochschule Hannover
Verified postcode
Hanover, Germany· Recruiting
University Children's Hospital Schleswig-Holstein
Verified postcode
Lübeck, Germany· Recruiting

Common questions

What is the main goal of this study?

The main goal is to understand how lung problems caused by cystic fibrosis (CF) start and develop in babies and young children, especially those diagnosed early.

Will my child need extra tests for this study?

Mostly, the study will use information from your child's routine hospital check-ups. You will also have monthly phone calls and quarterly questionnaires about their health.

Who can join this study?

Children recently diagnosed with CF can join, including those diagnosed through newborn screening or later in childhood due to symptoms, as long as their diagnosis is confirmed.

What if my child has other health problems?

Children with other significant health issues, especially other long-term lung conditions, may not be able to join the study.

Can I change my mind about participating?

Yes, you can withdraw your child from the study at any time without it affecting their medical care.

How to find out more

Marcus A Mall, MD

marcus.mall@med.uni-heidelberg.de +49 6221 56 4502 Official trial record

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