A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
This study is looking into a new combination medicine for very young children with Cystic Fibrosis, specifically those aged 12 months up to two years old. The main goal is to check if the medicine is safe and that their bodies handle it well. Researchers will closely monitor for any side effects and see how the medicine works in the body. They will also measure changes in sweat chloride, which is a key indicator for Cystic Fibrosis. This is a Phase 3 study, meaning it's a key step to see if this treatment could eventually become available to patients.
At a glance
What is this study about?
Cystic Fibrosis (CF) is a condition that affects many parts of the body, especially the lungs and digestive system. It's caused by a faulty gene that controls the movement of salt and water in and out of cells.
This study is testing a new combination medicine for children between 12 months and two years old who have CF. This medicine is already approved for older children and adults, and now researchers want to see if it's safe and works well for this younger age group. The medicine aims to help the faulty protein in CF work better, which can improve the symptoms.
The main things researchers will be checking are the safety of the medicine and how well the children’s bodies handle it. They will also look at how the medicine is absorbed and processed by the body, and if it helps to reduce the amount of salt in the children's sweat, which is a good sign the treatment is working.
Key takeaways
- This study is for babies and toddlers with Cystic Fibrosis, aged 12 to 24 months.
- It aims to check the safety and how well a new combination medicine is handled by the body.
- The medicine is given as granules that can be mixed with food.
- Children will have regular medical checks, including blood tests and heart monitoring.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
Anyone aged between 12 months and two years old with Cystic Fibrosis might be able to take part in this study. Both boys and girls are welcome to consider participating.
The research team will need to check your child's specific type of Cystic Fibrosis and their overall health to make sure the study is a good fit. This helps ensure the study is safe and that the results will be accurate.
- Is my child aged between 12 months and 2 years old?
- Does my child have a confirmed diagnosis of Cystic Fibrosis?
- Is my child generally healthy enough to participate in a study?
- Am I able to commit to the required clinic visits for my child?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If your child takes part, they will receive the study medicine, which will be in a granule form that can be mixed into food. They will have several visits to the clinic where doctors and nurses will monitor their health closely. These visits will involve checks like blood tests, measurements of their heart activity using an ECG, checking their breathing and oxygen levels, and taking their temperature and blood pressure. They will also have sweat tests to measure chloride levels. Your child will be monitored for any side effects throughout the study, which will last for a certain period, followed by ongoing checks.
Potential risks and benefits
Locations (3)
- —Germany
- —Netherlands
- —Denmark
Common questions
What is Cystic Fibrosis?
Cystic Fibrosis (CF) is a genetic condition that mainly affects the lungs and digestion. It causes thick, sticky mucus to build up in the body.
What is a 'Phase 3' study?
A Phase 3 study means the medicine has already gone through earlier safety checks and is now being tested on a larger group to confirm it is safe and effective.
What do the different medicines like VX-770 and Kalydeco do?
These are different names for types of medicines used to treat Cystic Fibrosis. The study is using a combination of these to see if they work well together for young children.
What is 'sweat chloride' and why is it measured?
People with CF often have high levels of salt in their sweat. Measuring sweat chloride helps doctors understand how severe the CF is and if treatments are working.
Will my child definitely get the new medicine?
Yes, in this study all participants will receive the investigational medicine. There is no placebo group.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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