About the BEACON-CF clinical research study

The purpose of the clinical research study

The BEACON-CF clinical research study is exploring how safe, tolerable, and effective an investigational study drug called VX-522 is for people with cystic fibrosis who are not expected to benefit from CFTR modulators.

What happens during the clinical research study

How the study will work

There are two parts to the BEACON-CF clinical research study:

Part 1 is exploring the safety and tolerability of a single dose of VX-522 for people with cystic fibrosis who are not expected to benefit from CFTR modulators.
Part 2 will help to find out if multiple doses of VX-522 are safe, well tolerated, and work as a potential treatment for people living with cystic fibrosis who are not expected to benefit from CFTR modulators.

The presented study specifics may be subject to amendments.

Study tests and procedures

Alongside receiving the investigational study drug, you will take part in other tests and procedures throughout the clinical research study. For example, this may include:

Questions about yourself and your health
A physical exam
A lung function test called spirometry, with the option to undergo a lung examination called bronchoscopy at specific clinical research sites
Measurements of your heart activity
Blood and urine samples

Full details of the tests and procedures involved will be explained to you before you begin the clinical research study.

Who can take part

You may be able to take part in this clinical research study if you:

Have been diagnosed with cystic fibrosis and are not expected to respond to CFTR modulators
Have a forced expiratory volume in 1 second (FEV1) value of more than or equal to 40% for Part 1 of the study, or more than or equal to 40% and less than or equal to 90% for Part 2
Do not have a history of solid organ or hematological transplantation
Are between 18 and 65 years old

Only people with a certain type of change in the CFTR gene are able to take part in this study. The study team will do a genetic test to confirm that you meet the requirements to take part.

Interest in this clinical research study is welcomed from all people who meet the requirements of the study, regardless of sex, gender, sexual orientation, race, or religion. Everyone who takes part will be helping to increase the knowledge of cystic fibrosis and how it is treated.

Study recruitment video

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