Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
This study is investigating a medication combination coded as ELX/TEZ/IVA for people with cystic fibrosis (CF). This treatment is for individuals who have CF but do not have the most common genetic change, called F508del, which affects the CFTR gene. The main aim is to see how safe the medication is and how well it helps people feel better and live healthier lives over a long period. Researchers will also look at how the medicine works in the body. This is a follow-on study for people who have already taken part in a previous trial, meaning they have some experience with these medicines. It’s part of a bigger effort to find effective treatments for all people with CF.
At a glance
What is this study about?
Cystic fibrosis (CF) is a condition that mainly affects the lungs and digestive system. It's caused by faulty genes. For many years, scientists have been working on new medicines that can fix some of these genetic problems, making a big difference for people with CF.
This particular study is focusing on a specific new treatment known as ELX/TEZ/IVA. What's special about this study is that it's for people with CF who don't have the most common genetic fault, called F508del. While some excellent treatments already exist for those with F508del, finding effective options for others is really important. This research aims to see if ELX/TEZ/IVA can help improve the health and well-being of these individuals.
The study will carefully follow volunteers over a long time to understand if the treatment is safe to use and if it effectively helps manage their CF symptoms. Researchers will also learn more about how the medicine works in the body. The goal is to provide more evidence that this treatment could be a safe and useful option for a wider group of people living with cystic fibrosis.
Key takeaways
- This study is for people with CF who do not have the F508del gene change.
- It evaluates the long-term safety and effectiveness of ELX/TEZ/IVA (and IVA).
- Only people who completed a previous study with these medicines can join.
- The study aims to expand treatment options for more people with CF.
- Participation involves ongoing medication and regular health checks.
Who may be eligible?
This study is for adults and children aged 6 years and older who have cystic fibrosis. To join, you would have already taken part in a previous study involving these same medicines (ELX/TEZ/IVA or IVA alone). This means you would have completed the treatment part of that earlier study, even if you had some short breaks from the medicine.
However, you won't be able to join if you had any bad reactions or couldn't tolerate the study medicine in the previous trial. The research team will also check for other specific health details to make sure the study is a safe and good fit for you.
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.
- I am 6 years old or older.
- I have cystic fibrosis (CF).
- I do not have the F508del gene change in my CF diagnosis.
- I previously took part in another study with ELX/TEZ/IVA or IVA and completed the treatment part of it.
- I did not have any serious problems with the study medicines in that previous trial.
What does participation involve?
If you join this study, you will continue to take the study medication (ELX/TEZ/IVA or IVA) as prescribed. You will have regular visits to the clinic, where the study team will carry out various health checks. These checks might include blood tests, lung function tests, and discussions about how you are feeling and any side effects you might be experiencing. The study is designed to follow participants over a long period, but the total duration for you would depend on how long you were in the previous study and the specific schedule set by the researchers. You’ll be carefully monitored throughout your participation.
Potential risks and benefits
Locations (81)
- Medizinische Universität Innsbruck - Heilstättenschule UniversitätsklinikVerified postcodeInnsbruck, Austria
- CUB Hôpital ErasmeVerified postcodeBrussels, Belgium
- UZ Brussel - Campus JetteVerified postcodeBrussels, Belgium
- Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University HospitalVerified postcodeEdegem, Belgium
- Universitair Ziekenhuis GentVerified postcodeGhent, Belgium
- Universitaire Ziekenhuizen Leuven - Campus GasthuisbergVerified postcodeLeuven, Belgium
- Universite Catholique de Louvain - PulmonologyVerified postcodeWoluwe-Saint-Lambert, Belgium
- Stollery Children's HospitalVerified postcodeEdmonton, Canada
- Le Centre de recherche du CHUM (CRCHUM)Verified postcodeMontreal, Canada
- McGill University Health Centre, Glen Site, Montreal Children's HospitalVerified postcodeMontreal, Canada
- Hospital for Sick Children - PulmonologyVerified postcodeToronto, Canada
- British Columbia Children's HospitalVerified postcodeVancouver, Canada
Common questions
What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic condition that mainly affects the lungs and digestive system, making mucus thick and sticky.
What does F508del mean?
F508del is the most common gene change that causes cystic fibrosis. This study is for people who don't have this specific change.
What medicines are being studied?
The study medicines are called ELX/TEZ/IVA (a combination) and IVA alone. They are designed to help the faulty protein in CF work better.
Will I have to pay to be in the study?
No, you will not have to pay for the study medication or any study-related tests and appointments.
Can I stop taking part if I change my mind?
Yes, you are free to withdraw from the study at any time without giving a reason, and it will not affect your future medical care.
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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