Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
This study, called STOP PEDS, is for children and young people aged 3 to 18 with Cystic Fibrosis (CF). We want to find the best way to treat lung flare-ups (called pulmonary exacerbations) in CF. The study compares two methods: one group gets antibiotics immediately when a flare-up starts, while the other group only gets antibiotics if their condition gets worse. We want to see if we can safely reduce the amount of antibiotics children take over a year, as this is important for their long-term health. The study also looks at how safe and effective each approach is, both in the short-term and over a year. There are also optional smaller studies, like collecting throat swabs or monitoring lung function at home.
At a glance
What is this study about?
When children and young people with Cystic Fibrosis (CF) get a lung flare-up, called a pulmonary exacerbation, healthcare professionals currently have different ideas about the best way to treat it. Some believe it's best to start antibiotics straight away, while others think it's better to wait and see if symptoms improve on their own, only using antibiotics if things get worse. The STOP PEDS study aims to find out which of these approaches is safer and more effective for children with CF.
This study is important because treating flare-ups quickly and effectively helps children with CF stay as healthy as possible. However, using antibiotics too often can lead to problems like antibiotic resistance, where the medicines stop working as well over time. This study wants to see if a 'watch and wait' approach, where antibiotics are only given if truly needed, can help reduce the total amount of antibiotics children take, while still keeping them well.
The researchers will follow two main groups of children. One group will start 14 days of antibiotics as soon as a flare-up begins. The other group will try to manage their symptoms by increasing their airway clearance (special breathing exercises) and only start antibiotics if their symptoms get worse or don't improve after a certain time. By comparing these two strategies, the study hopes to give clearer guidance to families and doctors on the best way to manage these common flare-ups.
Key takeaways
- Study compares two ways to treat lung flare-ups in children with CF.
- 'Immediate antibiotics' vs. 'tailored treatment' (antibiotics only if needed).
- Aims to reduce antibiotic use and find the safest, most effective approach.
- Participation lasts for one year, with regular monitoring.
- Optional extra studies like home testing are available.
Who may be eligible?
This study is looking for children and young people with Cystic Fibrosis (CF). Generally, they need to be between 3 and 18 years old. To join the main part of the study, participants must be taking a highly effective CF medicine (called HEMT) for at least three months.
We also have smaller groups for children aged 3 to 5 on HEMT, and for those aged 6 to 18 who are not taking HEMT. All participants must have a confirmed CF diagnosis based on specific tests, and they, or their parents/guardians, must understand and agree to take part.
There are also a few reasons someone might not be able to join. For example, if they are already taking antibiotics or certain other medications, or have a health issue that might make it unsafe to take part, they wouldn't be eligible. They also need to be able to use a mobile phone for text messages and have internet access.
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.
- Is my child aged between 3 and 18 years old?
- Does my child have a confirmed diagnosis of Cystic Fibrosis?
- Is my child currently taking a highly effective CF medicine (HEMT) for at least 3 months (for main group)?
- Does my child have access to a mobile phone for texts and the internet?
- Is my child currently healthy and not on active antibiotics or certain other medications?
What does participation involve?
If you join this study, your involvement will typically last for one year. You will continue to see your regular CF doctors, but the study will guide how your lung flare-ups are treated. You'll be put into one of two groups by chance: either starting antibiotics immediately when a flare-up begins, or only starting them if your symptoms worsen. You'll stick to this treatment plan for all flare-ups during your year in the study.
Throughout the year, the study team will keep in touch to monitor your health. This might involve your regular clinic visits, answering questionnaires, and potentially some basic home monitoring if you choose to take part in optional sub-studies. Some optional parts of the study include providing throat or nose swabs, or monitoring your lung function and vital signs at home using special equipment. The total number of clinic visits will generally be what you already have planned with your CF team, plus any additional checks needed for a flare-up. You or your child will continue to take any other medicines for CF as normal.
Potential risks and benefits
Locations (33)
- The Children's Hospital Alabama & University of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- Tucson Cystic Fibrosis CenterVerified postcodeTucson, United States· Recruiting
- Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis ClinicVerified postcodeLos Angeles, United States· Recruiting
- Stanford UniversityVerified postcodePalo Alto, United States· Recruiting
- Rady Children's Hospital at University of California San DiegoVerified postcodeSan Diego, United States· Not yet recruiting
- Children's Hospital of ColoradoVerified postcodeAurora, United States· Recruiting
- Children's Healthcare of Atlanta & Emory UniversityVerified postcodeAtlanta, United States· Recruiting
- Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern UniversityVerified postcodeChicago, United States· Not yet recruiting
- Riley Hospital for Children & Indiana UniversityVerified postcodeIndianapolis, United States· Recruiting
- University of IowaVerified postcodeIowa City, United States· Recruiting
- Johns Hopkins Hospital, Johns Hopkins UniversityVerified postcodeBaltimore, United States· Recruiting
- Boston Children's Hospital & Harvard UniversityVerified postcodeBoston, United States· Recruiting
Common questions
What is a 'pulmonary exacerbation'?
It's a medical term for a lung flare-up in people with Cystic Fibrosis, meaning their breathing symptoms get worse.
What does 'HEMT' mean?
HEMT stands for Highly Effective Modulator Therapy. These are modern medicines for CF that help improve how the body works.
Will I or my child still get regular CF care?
Yes, you will continue to receive all your normal CF care from your usual healthcare team while in the study.
What happens if my child's symptoms get worse in the 'tailored treatment' group?
If specific signs of worsening symptoms or failure to improve are met, your child will then start antibiotics, as planned in the study design.
Can I choose which treatment group my child is in?
No, participants are randomly assigned to a group, like flipping a coin. This helps make the study results fair and unbiased.
How to find out more
Erika Enright
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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