Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
This research is a continuation study focusing on the safety and effectiveness of a combination medicine, VNZ/TEZ/D-IVA, for people with cystic fibrosis (CF). It’s for individuals who are one year old or older and have already taken part in a previous related study about this medicine. The main goal is to understand how well the medicine works and if it has any long-term effects, both positive and negative. By carefully tracking participants over time, researchers hope to learn more about how this treatment can help manage CF and improve lives.
At a glance
What is this study about?
This study is a follow-up to an earlier trial, and it's designed to keep a close eye on a new combination medicine for cystic fibrosis (CF) called VNZ/TEZ/D-IVA. Think of it like this: if you've already tried a new medicine in a first study, this second study watches you for longer to make sure it's still safe and continues to help you. The main reason for this research is to see how well the medicine works over a long period and if it has any side effects that might only show up after a while. This is really important for medicines like those for CF, which people often take for many years.
Cystic fibrosis is a condition that affects how the body makes mucus, sweat, and digestive juices. These fluids are usually thin and slippery, but in CF, they become thick and sticky, which can block tubes, ducts, and passageways, especially in the lungs and pancreas. This can lead to breathing problems, infections, and difficulties absorbing food. Medicines like VNZ/TEZ/D-IVA aim to correct some of the underlying problems that cause CF.
By taking part in this long-term study, you would be helping doctors and scientists understand the full picture of how this new treatment works. This information is critical for deciding if the medicine should become widely available and for helping future patients with CF lead healthier lives.
Key takeaways
- This is a long-term follow-up study for a CF medicine.
- It's for people aged 1 and older who were in a previous study.
- The study looks at long-term safety and how well the medicine works.
- Your health will be closely monitored with regular visits.
- You have the right to stop participating at any time.
Who may be eligible?
To be considered for this study, you must have already taken part in a previous related study called VX21-121-105. This study is mainly designed for people who completed the treatment in that earlier study. If you had some breaks in taking your medicine during the first part of that previous study but didn't permanently stop, and you completed all your visits, you might still be able to join.
There are also some reasons why you might not be able to join. For example, if you have severe liver problems, especially those linked with high blood pressure in the liver, or if you've had an organ transplant or certain types of cancer in the past, you wouldn't be able to take part. Also, if you experienced serious problems or couldn't tolerate the study medicine during the previous study, you won't be eligible for this one.
The study team will carefully review your full medical history with you to make sure it's safe for you to take part and that you meet all the necessary requirements.
- I was in the previous study (VX21-121-105).
- I completed the treatment phase of that previous study (or had interruptions but completed all visits).
- I do not have severe liver problems.
- I have not had an organ transplant or specific types of cancer.
- I did not have serious drug intolerance in the previous study.
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you'll continue to take the study medicine, VNZ/TEZ/D-IVA, as prescribed. You'll have regular visits to the clinic where the study team will monitor your health. These visits will involve various assessments, such as blood tests, urine tests, and checks of your breathing and other body functions, to see how the medicine is working and if you're experiencing any side effects. The study team will provide you with the medication and precise instructions on how to take it.
The study is designed to be a long-term follow-up to a previous study you participated in. The specific number and frequency of visits, as well as the total duration you will be in the study, will be discussed in detail by the study team. They will ensure you understand what's involved every step of the way.
Potential risks and benefits
Locations (37)
- Children's Hospital of Orange CountyOrange, United States
- Stanford University - Palo Alto - PulmonologyPalo Alto, United States
- Children's Hospital of Colorado - PulmonologyAurora, United States
- The Emory Clinic - Clifton Road - PulmonologyAtlanta, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyChicago, United States
- JW Riley Hospital for Children - PulmonologyIndianapolis, United States
- Boston Children's HospitalBoston, United States
- Children's Health Care d/b/a Children's Hospitals and Clinics of MinnesotaMinneapolis, United States
- The Children's Mercy HospitalKansas City, United States
- St. Louis Children's Hospital - PulmonologySt Louis, United States
- Cohen Children's Medical Center - Lakeville RoadLake Success, United States
- Cincinnati Children's Hospital Medical Center - PulmonologyCincinnati, United States
+25 more sites — see the official record for the full list.
Common questions
What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic condition that causes thick, sticky mucus to build up in the body, especially in the lungs and digestive system.
What is VNZ/TEZ/D-IVA?
It's a combination medicine being studied to treat cystic fibrosis. It's designed to help correct some of the underlying problems that cause CF.
Why is this study important?
This study helps us understand how safe and effective the new medicine is over a long time, which is crucial for people who take CF medicines for many years.
Do I have to pay to be in the study?
No, you do not have to pay for the study medicine or the study-related tests and appointments. These are usually covered by the study sponsor.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time, for any reason, and it won't affect your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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