A Phase 1/2 Dose-escalation Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
This clinical trial is looking for adult volunteers (18 years and older) with cystic fibrosis (CF). It's for people whose CF is caused by a specific genetic change that doesn't respond to the usual CF medicines (CFTR modulators). The study is testing a new inhaled medication called VX-522. This is an early-stage trial (Phase 1/2), which means researchers are carefully checking how safe the new medicine is and if people can tolerate it. They also want to see if it starts to make a difference to their health. Participants will use VX-522 with a nebuliser, and some may also take another CF medicine called Kalydeco. The study involves regular check-ups to monitor a person’s overall health, lung function, and how their body reacts to the new treatment.
At a glance
What is this study about?
This study is investigating a new medicine called VX-522 for adults with cystic fibrosis (CF). CF is a genetic condition that mainly affects the lungs, making it hard to breathe, and can also impact other parts of the body. Many people with CF benefit from treatments called CFTR modulators, but not everyone has the specific genetic make-up that responds to these medicines. This study is specifically for those adults with CF whose genes mean these standard CFTR modulator treatments aren't suitable or effective for them.
The main goal of this trial is to see how safe the new medicine, VX-522, is and whether patients can comfortably take it. As it's an early-stage study (called Phase I and II), it means this medicine hasn't been widely tested in people before. Researchers will be giving different doses to find the safest and most effective amount. They will closely monitor participants for any side effects and check various health measures to ensure their well-being.
Researchers will also be looking to see if VX-522 starts to show any positive effects. They’ll check lung function, which is a key indicator of CF health, and other health markers during the trial. This is a crucial step in developing new treatments, offering hope for individuals with CF who currently have limited options.
Key takeaways
- This study is for adults (18+) with cystic fibrosis.
- It's for those whose CF doesn't respond to standard CFTR modulator medicines.
- The study tests an inhaled medicine called VX-522.
- It's an early-stage trial focusing on safety, tolerance, and early signs of benefit.
- Regular health checks and monitoring are a key part of participating.
Who may be eligible?
This study is looking for volunteers who are adults, meaning they must be 18 years old or older. There is no upper age limit for taking part.
Both men and women are welcome to participate in this study. The most important requirement, however, is that you have cystic fibrosis, and specifically, your type of CF must be one that doesn't usually respond to the common CF treatments called CFTR modulators. Your doctor will be able to tell you if this applies to you.
There might be other specific health requirements for joining, which the study team will explain. This is to ensure the study is safe for everyone involved and that the results are clear.
- Are you 18 years old or older?
- Do you have cystic fibrosis?
- Has your doctor told you that your CF type does not respond to common CFTR modulator medicines?
- Are you willing to attend regular appointments and health checks?
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, you would receive the new medicine, VX-522, through a nebuliser, which means you would breathe it in as a fine mist. Some participants might also take Kalydeco tablets alongside it. Throughout the study, you'll have regular visits where the team will perform various health checks. These include standard tests like blood and urine samples, as well as heart checks (ECGs) and monitoring your breathing (spirometry).
They'll also record your vital signs (such as blood pressure and heart rate) and measure the oxygen levels in your blood. These check-ups are very important for closely monitoring your safety and how your body is reacting to the new medicine. The study will involve regular assessments up to an 8-week period, with further safety follow-ups after that. The study team will tell you the total duration of your involvement.
Potential risks and benefits
Locations (6)
- —Germany
- —Italy
- —Netherlands
- —Belgium
- —Sweden
- —Spain
Common questions
What is VX-522?
VX-522 is a new medicine being developed for cystic fibrosis. It's given through a nebuliser, meaning you breathe it in.
Why is this study only for people whose CF doesn't respond to other medicines?
This study focuses on people with specific types of CF where current treatments like CFTR modulators aren't effective. It aims to find new options for them.
What's the difference between Phase 1 and Phase 2?
Phase 1 is mainly about checking the safety of a new medicine and finding the right dose. Phase 2 starts to look more closely at how well the medicine works, while still monitoring safety.
Will I know if I'm getting the active medicine or a dummy treatment?
In some studies, participants might not know. The study team will explain the details of this specific trial's design before you decide to join.
How long will I be in the study if I participate?
The study involves regular check-ups for at least 8 weeks, with further safety follow-ups. The study team will give you a full timeline.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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