A Phase 3 Open-label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
This long-term study is investigating a combination medicine called Kaftrio, or by its full name, Elexacaftor/Tezacaftor/Ivacaftor, for individuals aged 2 and above who have Cystic Fibrosis. The main goal is to carefully check its safety and how well people tolerate it by monitoring for any side effects, changes in blood tests, heart readings, and other important health measures. Researchers also want to see if the medicine can improve key indicators of Cystic Fibrosis, such as the amount of salt in sweat and how well the lungs are working. This research is in its final stages before the medicine might become widely available.
At a glance
What is this study about?
This study is a research project designed to learn more about a medicine called Kaftrio, also known by its longer name, Elexacaftor/Tezacaftor/Ivacaftor. Kaftrio is a treatment for Cystic Fibrosis (CF), a condition that affects the lungs and digestive system.
The main purpose of this study is to look at the long-term safety of Kaftrio for people with CF who are 2 years old and over. This means the researchers want to understand if there are any side effects or health concerns that might show up over a longer period of time. They will also be checking how well people’s bodies react to the medicine.
Besides safety, the study will also look at how well Kaftrio works for people with CF. This includes seeing if it can help improve things like the amount of salt in sweat (which is often higher in people with CF) and how well a person's lungs are working. By studying these factors, the research aims to provide a clear picture of Kaftrio's overall benefits and any potential drawbacks for people living with Cystic Fibrosis.
Key takeaways
- This study evaluates Kaftrio for people with Cystic Fibrosis.
- It's looking at the long-term safety and effectiveness of the medicine.
- Participants will be aged 2 years and older.
- It's an 'open-label' study, meaning all participants receive an active drug.
- Includes regular health checks like blood tests and lung function tests.
- Aims to improve care for people with Cystic Fibrosis.
Who may be eligible?
This study is open to anyone of any age, male or female, who has Cystic Fibrosis. This means that if you have been diagnosed with Cystic Fibrosis, you might be able to take part, whether you are a young child or an adult.
However, even though the age range is wide, there will be more detailed health checks and specific requirements that a doctor will need to go through with you to ensure the study is right for you. They will need to confirm that participating in this particular study is safe and appropriate for your individual health situation.
It's important to discuss your full medical history with the study team, as certain other health conditions or medications might affect whether you can join, even if you meet the general age and diagnosis criteria.
- Do I have Cystic Fibrosis?
- Am I (or the person I care for) 2 years old or older?
- Am I able to attend regular clinic appointments?
- Am I willing to take the study medication as prescribed?
- Do I have any other significant health conditions or medications that the study team should be aware of?
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 would be taking one of the study medicines, which could include Kaftrio or Kalydeco, either as granules (small grains to mix with food) or as tablets. The medicine you receive will be carefully chosen by the study team.
You will have regular visits to the clinic or hospital, where the study team will carry out various tests. These include blood tests to check your overall health, heart checks using an ECG (a simple test to look at your heart's electrical activity), measurements of your vital signs like blood pressure and temperature, and a check of your oxygen levels using a small clip on your finger. They will also ask you how you are feeling and if you have experienced any new symptoms or side effects.
Throughout the study, the team will closely monitor your health to make sure the medicine is safe and if it is helping with your Cystic Fibrosis. They will also look at specific measures related to CF, such as sweat tests and special breathing tests to measure how well your lungs are clearing air. The total duration of your participation in the study will be explained in detail by the study team, including how long you'll be taking the medicine and how often you'll have follow-up appointments.
Potential risks and benefits
Locations (1)
- —Germany
Common questions
What is Cystic Fibrosis?
Cystic Fibrosis is a genetic condition that mainly affects the lungs and digestive system by causing thick, sticky mucus to block airways and organs.
What is Kaftrio?
Kaftrio is a medicine for Cystic Fibrosis. It’s a combination of three different drugs that work together to help the body’s cells work better.
What will researchers be looking for in this study?
They will primarily check for the safety of Kaftrio over a long time, looking for side effects, and also measure how well it improves lung function and other CF symptoms.
Will I get the actual medicine or a dummy pill?
This is an 'open-label' study, which means everyone who takes part will receive one of the active study medicines, not a dummy pill (placebo).
Who is funding this research?
This information is not provided in the summary, but it's a good question to ask the study team if you are interested in joining.
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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