A study to evaluate a new mRNA therapy in adults with CF mutations that don’t respond to CFTR modulator therapies (VX21-522-001)
- Therapeutic approach
- Genetic Therapy
- Trial status
- Open to recruitment Participating Centres
- Trials Tracker ID
- Last updated
A phase 1 study to evaluate the safety, tolerability and effectiveness of VX-522 mRNA therapy in people with CF aged 18 and over with CFTR mutations that don’t respond to CFTR modulator therapies.Study details
mRNA therapy is a new type of treatment for people with CF, to be assessed in clinical trials.
If you are aged between 18 and 65 and have CF mutations that mean you can’t take CFTR modulator treatments, you may be eligible to take part in this study. Example CF mutations could include Class I mutations (where no CFTR protein is produced), nonsense mutations (like G542X and W1282X), and some types of splicing mutations. Talk to your CF team if you don’t know which CF mutations you have or if you think you might be eligible.
This study – also referred to as the BEACON-CF clinical research study – is exploring the safety and tolerability of a single dose of VX-522 (an inhaled mRNA therapy) for people with cystic fibrosis who are not expected to benefit from CFTR modulators.
Participants will be given a single dose of VX-522, which they will inhale through a nebuliser.
Participants will be asked to take part in daily monitoring visits for the first week. They will also be asked to attend follow-up visits on day 14, day 28, week 12, and week 24.
Please note that you do not need to be receiving your usual care from one of the recruiting centres to be able to take part in the trial. Please speak to your local CF team or use the ‘Get in touch’ button opposite if you are interested in taking part.
- Phase 1
- Length of participation
- 28 weeks
- CF sponsor
Who can take part?
- Age range
- Between 18 and 65 years
- Including people
With two CFTR gene mutations that are not responsive to CFTR modulator treatments like Kaftrio, Orkambi and Symkevi
FEV1 greater than or equal to 40% of predicted mean for age, sex, and height
Total body weight greater than 50 kg
- Excluding people
With a history of uncontrolled asthma within a year prior to screening
History of solid organ or hematological transplantation
Talk to your CF team if you’re not sure whether you’re eligible.
Get in touch