A study to evaluate a new mRNA therapy in adults with CF mutations that don’t respond to CFTR modulator therapies (VX21-522-001)

An early phase study to evaluate the safety, tolerability and effectiveness of a new mRNA therapy (VX-522) in people with CF aged 18 and over with CFTR mutations that don’t respond to CFTR modulator therapies (VX21-522-001)

Vertex has made the decision to stop the investigation of the potential cystic fibrosis gene mRNA therapy (VX-522) and therefore terminated the VX21-522-001 trial. Despite efforts to improve tolerability of the multiple-ascending dose (MAD) portion of the study, including corticosteroids and reducing VX-522 dose, tolerability issues remained; Vertex has decided to not support the continuation of this study.

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.

Find out more about  genetic therapies and what’s involved in genetic therapy clinical trials.

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.