Active not recruitingPHASE3INTERVENTIONAL

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

This research is a continuation of previous studies investigating a new medication called VX-121/TEZ/D-IVA for people living with cystic fibrosis. The main goal is to check its long-term safety, how well people tolerate it, and how effective it is over a longer time. This study is for participants who have recently taken part in earlier studies of this specific drug combination. By continuing to follow these patients, researchers hope to gain a better understanding of the treatment's ongoing effects and its potential to improve the health of people with cystic fibrosis. It will help confirm if the benefits seen so far continue in the long run.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

822

Start

08 Nov 2022

Estimated completion

30 Oct 2026

Cystic Fibrosis

What is this study about?

This study is all about understanding a new medicine called VX-121/TEZ/D-IVA, which is being developed to help people with cystic fibrosis. Cystic fibrosis is a long-term condition that affects several organs in the body, especially the lungs, making it hard to breathe and prone to infections. Treatments like this aim to improve lung function and overall health.

Researchers want to see how safe and helpful this new combination medicine is over a longer period. Many medicines are first tested for shorter times, but it's important to know what happens when people take them for many months or even years. This study helps answer those questions by carefully observing people who have already been taking the medicine in earlier research studies.

The information gathered from this study will be very important for doctors and patients. It will help them understand the full picture of this treatment – not just if it works in the short term, but also if it remains safe and effective for ongoing use, which is crucial for managing a long-term condition like cystic fibrosis. This is the final stage of testing before a medicine can be approved for wider use.

Key takeaways

This study is for people with cystic fibrosis aged 12 and older.
It's testing a combination medicine called VX-121/TEZ/D-IVA.
The main goal is to check its long-term safety and how well it works.
You must have been in a specific previous study for this medicine to join.
It's a long-term follow-up to see continued effects.

Who may be eligible?

This study is designed for adults and young people aged 12 and older who have cystic fibrosis. To join, you must have previously taken part in one of two specific earlier studies (known as VX20-121-102 or VX20-121-103) that also tested this same medicine.

Basically, if you finished your treatment in one of those previous trials, or if you had a short break from the medicine but then completed your past study visits, you might be able to join this new study. The study wants to continue following people who already have experience with this particular treatment.

However, you won't be able to join if you had a bad reaction to the medicine in a previous study that made you stop taking it permanently. Also, if you are pregnant or breastfeeding, you cannot take part. Your study doctor will check other important details to see if this study is right for you.

Quick self-check

Are you 12 years old or older with cystic fibrosis?
Did you complete treatment in a previous study called VX20-121-102 or VX20-121-103?
Did you not have a serious, permanent problem with the study medicine in the past?
Are you not currently pregnant or breastfeeding?

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 will continue to receive the VX-121/TEZ/D-IVA combination medicine, just as you did in the previous studies. You'll have regular visits to your study clinic, where the research team will monitor your health. These visits will involve check-ups, some tests, and discussions about how you're feeling and any effects you might be experiencing from the medicine.

The duration of your participation will depend on the study design, but it's focused on understanding long-term effects, so it will last for an extended period. The research team will explain exactly what each visit involves and how long the study is expected to run, ensuring you understand the full commitment before you start.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of continuing in this study is that you would keep receiving a medication that has already shown promise in previous research for cystic fibrosis. This could mean continued improvement in your health. However, like all medicines, VX-121/TEZ/D-IVA can have side effects, which might be new or become more noticeable over a longer period. The research team will carefully monitor you for these. It’s important to remember that joining a study is always your choice, and you are free to withdraw at any time if you change your mind, without affecting your regular medical care.

Locations (195)

University of Alabama at Birmingham
Birmingham, United States
Banner University of Arizona Medical Center
Tucson, United States
University of Arkansas for Medical Sciences
Little Rock, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, United States
Children's Hospital Los Angeles
Los Angeles, United States
Kaiser Permanente
Oakland, United States
University of California Davis Medical Center
Sacramento, United States
University of California San Francisco, Lung Transplant Program
San Francisco, United States
Children's Hospital of Colorado
Aurora, United States
National Jewish Health
Denver, United States
University of Florida, Shands Hospital
Gainesville, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
Hollywood, United States

+183 more sites — see the official record for the full list.

Common questions

What is cystic fibrosis?

Cystic fibrosis is a genetic condition that mainly affects the lungs and digestive system, making mucus thick and sticky, which can cause breathing problems and infections.

What is a 'combination therapy'?

A combination therapy means using several different medicines together. In this study, it's VX-121, tezacaftor, and deutivacaftor all at once.

Why is this study only for people who were in previous studies?

This study is a continuation to see how the medicine works in the long term for people who have already started taking it in earlier research. It helps track their progress over time.

What does 'Phase 3' mean for a study?

Phase 3 is usually the final stage of testing a new medicine in a large group of people to confirm its safety and effectiveness before it can be approved for general use.

Who can I talk to if I have more questions?

You should always talk to your doctor or the study team if you have any questions about this research or your health.