Active not recruitingPHASE3INTERVENTIONAL

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

This research study is checking the long-term effects of a treatment called ELX/TEZ/IVA for people with cystic fibrosis. It's for patients who are 12 months old and older and have already taken part in a related study (called VX22-445-122 Part B). The main aim is to see how safe the medicine is over a longer period, how well people tolerate it, and if it continues to help with their cystic fibrosis symptoms. Researchers will also look at how the medicine works in the body. This is a follow-up study, meaning it builds on previous research, to gather more information about this important treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

Start

21 Nov 2024

Estimated completion

30 Sep 2027

Cystic Fibrosis

What is this study about?

This study is looking into a treatment called ELX/TEZ/IVA for people who have cystic fibrosis. Cystic fibrosis is a condition that mainly affects the lungs and digestive system, making it hard to breathe and digest food properly. While treatments like ELX/TEZ/IVA are already being used, doctors and scientists want to understand more about their long-term effects.

The main goal of this particular study is to see how safe and effective ELX/TEZ/IVA is over a longer period. This means checking if it continues to help with cystic fibrosis symptoms, if people tolerate it well, and if there are any new side effects that only appear after taking the medicine for a long time. It's important to gather as much information as possible to make sure treatments are as good and safe as they can be for patients.

This study is a follow-up for people who have previously taken part in another related study where they were already given this medicine. By continuing to observe these participants, researchers can get a much clearer picture of what it’s like to use ELX/TEZ/IVA for many months or even years. This long-term information is incredibly valuable for doctors and patients deciding on the best course of treatment.

Key takeaways

This study is for people with cystic fibrosis who are already taking ELX/TEZ/IVA.
It aims to understand the long-term safety and how well the treatment works.
Participants must have completed a previous study involving this medicine.
You will continue to receive the study medicine and have regular health check-ups.
All participation is voluntary, and you can withdraw at any time.
This research helps improve long-term care for cystic fibrosis patients.

Who may be eligible?

This study is for people who have already been taking the ELX/TEZ/IVA medicine in a previous study. You would need to have finished taking the medicine in that first study, or if you had a break, you must not have permanently stopped taking it and completed all your scheduled visits for that study.

However, you cannot join this new study if you had a bad reaction to the medicine in the previous study that could put your health at risk. Also, if you are currently taking part in another research study for a different experimental medicine, you would not be able to join this one.

There might be other specific rules not listed here that determine if you can join. The study team will check all your details carefully to make sure the study is a good fit for you.

Quick self-check

Have I taken part in the previous study called VX22-445-122 Part B?
Did I complete taking the medicine in that study, or if I had a break, did I complete all the visits?
Did I have any severe or risky reactions to the medicine in the previous study?
Am I currently taking part in another research study for a different experimental medicine?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

We don’t have all the details about what taking part in this specific follow-up study will involve, such as the exact number of visits or how long each visit will be. However, typically in studies like this, you can expect regular visits to a clinic for check-ups. During these visits, doctors and nurses will monitor your health, perform various tests (which could include blood tests, breathing tests, and physical examinations), and ask how you are feeling and if you've experienced any changes.

You will continue to take the ELX/TEZ/IVA medicine as instructed by the study team. They will carefully track any side effects or changes in your condition. The overall length of time you'll be part of this study will be decided by the researchers, but the aim is to gather long-term information, so it’s likely to last for a significant period. You will always be given clear information about what to expect before you agree to take part.

Potential risks and benefits

Participating in this study might offer you some potential benefits, such as continued access to the ELX/TEZ/IVA treatment and close monitoring of your health by a specialist team. However, like all medicines and studies, there are potential risks. You might experience side effects from the medication, some of which could be unknown at this stage, or there could be discomfort from study procedures like blood tests. It's also possible that the treatment might not work as expected for everyone. It's very important to remember that joining any study is completely your choice, and you are free to withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (18)

The Kids Research Institute Australia
Nedlands, Australia
The Royal Children's Hospital
Parkville, Australia
Queensland Children's Hospital
South Brisbane, Australia
The Hospital for Sick Children
Toronto, Canada
British Columbia Children's Hospital
Vancouver, Canada
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Charité Universitätsmedizin BerlinX
Berlin, Germany
Kinderklinik III, Abt. fur Pneumologie
Essen, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Erasmus Medical Center
Rotterdam, Netherlands
Inselspital - Universitaetsspital Bern
Bern, Switzerland
Kinderspital Zurich
Zurich, Switzerland

+6 more sites — see the official record for the full list.

Common questions

What is the medicine ELX/TEZ/IVA?

It's a treatment used for people with cystic fibrosis, designed to help improve lung function and overall health.

Why is this study needed if I've already taken this medicine?

This study aims to understand the long-term safety and effectiveness of the medicine, which means gathering information over an extended period after the initial study.

Who can join this follow-up study?

You can join if you successfully completed a previous study (VX22-445-122 Part B) where you took this medicine, and you didn't have severe problems with it.

What will I have to do if I join?

You will continue taking the medicine and attend regular check-up appointments where your health will be monitored, and tests may be done.

What if I change my mind about taking part?

You have the right to leave the study at any time, and you don't need to give a reason. Your usual medical care will not be affected.