Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

This research study, called a Phase 1/2 trial, is investigating a new treatment called VX-522 for people aged 18 to 65 living with cystic fibrosis (CF). VX-522 is a type of gene therapy designed to help the body produce a working protein that is faulty in CF. The study is specifically for individuals whose CF is caused by gene mistakes (mutations) that don't respond to current CF medicines. Researchers want to find out if VX-522 is safe to use, how well people tolerate it, and if it can improve their health. This early-stage study is crucial for developing potential new treatments for those with CF who currently have limited options.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

Start

27 Feb 2023

Estimated completion

09 Sep 2026

Cystic Fibrosis

What is this study about?

This study is looking into a new treatment called VX-522, which is an mRNA therapy. mRNA therapies are a modern type of medicine that essentially give your body instructions to make a specific protein. In cystic fibrosis (CF), a particular protein called CFTR doesn't work correctly, leading to health problems. VX-522 aims to help your body produce a functional CFTR protein, potentially improving your CF symptoms. This is an important step because it targets the underlying cause of CF in a new way.

This is a 'Phase 1/2' study, which means it's an early stage of research. Phase 1 studies mainly focus on checking if a new medicine is safe and what dose might be best. Phase 2 studies then look more closely at whether the medicine actually works and continues to be safe. By combining these phases, researchers can gather important information more efficiently.

The study is specifically for adults with CF whose genetic changes (mutations) do not respond to the CFTR modulator treatments currently available. These include mutations where the body doesn't produce any CFTR protein at all. Finding effective treatments for this group is a high priority in CF research, and VX-522 could represent a new pathway for managing their condition.

Key takeaways

This study is for adults (18-65) with cystic fibrosis.
It's testing a new mRNA treatment called VX-522.
It targets CF types that don't respond to current medications.
The main goals are to check the safety, how well it's tolerated, and if it helps.
Strict health criteria are in place to ensure participant safety.
Participation involves regular clinic visits and health assessments.

Who may be eligible?

To be considered for this study, you need to be an adult between 18 and 65 years old with cystic fibrosis. A key requirement is that your specific type of CF, based on your genetic tests, must not respond to the CFTR modulator medicines that are currently available. This typically means you have genetic changes that stop your body from making any of the CFTR protein. You would also need to be in a stable condition with your CF.

There are also some general health criteria. For example, your weight needs to be within a certain range. For some parts of the study, your lung function, measured by a test called FEV1, needs to be at a certain level – not too low, but also not extremely high if you're taking part in certain stages. This helps ensure your safety during the study.

However, you would not be able to join if you have experienced uncontrolled asthma recently, if you’ve had an organ transplant, or if you have certain liver problems. Your oxygen levels would also need to be good. These checks are in place to protect your health and ensure the study is suitable for you.

Quick self-check

Are you between 18 and 65 years old?
Do you have cystic fibrosis?
Do your current CF medicines not work well for your specific type of CF?
Do you not have a history of organ transplantation or certain liver problems?
Are your lungs generally stable and your oxygen levels good?

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

What does participation involve?

Taking part in this study would involve regular visits to the study clinic for check-ups and to receive the investigational medicine, VX-522. The exact number and frequency of visits will depend on which part of the study you are in. You would undergo various assessments, such as physical examinations, blood tests, and lung function tests, to monitor your health and see how the treatment is affecting you. You would be given the study drug, VX-522. After the main treatment period, there would likely be a follow-up period to continue monitoring your health and any effects of the medicine. The total duration of your involvement could vary, but clinical trials typically last for several months to a year or more.

Potential risks and benefits

Participating in this study might offer a potential benefit of accessing an investigational treatment, VX-522, that is not yet available to the public. This treatment could potentially improve your health condition, especially if current CF medicines are not effective for you. However, as with any new medicine, there are potential risks and side effects that are not fully known yet. These will be carefully monitored by the study team, and you will be informed of any known risks. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (26)

University of Alabama at Birmingham - Child Health Research Unit
Birmingham, United States
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, United States
Stanford University - Palo Alto - Pulmonology
Palo Alto, United States
National Jewish Health
Denver, United States
Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, United States
PAREXEL International - Baltimore
Baltimore, United States
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, United States
MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, United States
Boston Children's Hospital
Boston, United States
University of Minnesota -Pulmonology
Minneapolis, United States
St. Louis Children's Hospital - Pulmonology
St Louis, United States
UC Health Holmes Hospital
Cincinnati, United States

+14 more sites — see the official record for the full list.

Common questions

What exactly is VX-522?

VX-522 is a new type of treatment called an mRNA therapy. It's designed to give your body instructions to produce a working protein that is faulty in people with cystic fibrosis.

Why is this study only for people whose CF doesn't respond to other medicines?

This study is specifically tailored for individuals whose genetic type of CF doesn't benefit from existing treatments. It aims to offer a new potential option for this group.

Will I know if I'm getting the actual medicine or a placebo?

In some parts of clinical trials, participants might not know if they are receiving the study drug or a placebo (an inactive substance). The study team will explain the design to you.

What if my health changes during the study?

Your health will be closely monitored by the study team throughout the trial. If your health changes significantly, they will evaluate whether it's safe for you to continue participating.

How long will I need to be involved in the study?

The total time you'd be involved can vary. Detailed information about the length of participation, including treatment and follow-up, will be provided to potential participants.