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A study to learn more about a combination of CFTR modulator medicines in people with Cystic Fibrosis


Therapeutic category
the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status


Participating Centres
Full title

A study to evaluate the effectiveness and safety of VX 445/Tezacaftor/Ivacaftor in people with Cystic Fibrosis who have two copies of the F508del gene

This study is being done to learn more about a new medication called ‘VX-445’ when taken in combination with tezacaftor and ivacaftor. All three of these medications are known as CFTR modulators. CFTR modulators work to treat the underlying genetic cause of cystic fibrosis. This combination of medication is being assessed in people with Cystic Fibrosis who are 12 years and older and who are homozygous for the F508del mutation. The study will investigate how well these medications work and to check that they are safe to use. It is the hope that this combination of medications will have positive effects in some people with Cystic Fibrosis. Participants will be asked to come into the clinic at the recruitment centre 7-8 times over a 5 month period where study assessments will take place (blood tests, questionnaires, lung function etc.).

Trial Reference Number


Trial type


the name of the treatment or therapy being researched

CFTR Modulators

Last edited date

30 October 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?

Top inclusion criteria
  • 12 Years and older
  • Homozygous for the F508del mutation
  • FEV1 value ≥40% and ≤90%
Top exclusion criteria
  • Clinically significant cirrhosis with or without portal hypertension.
  • Lung infection

CF centres running this trial


Alder Hey Children's Hospital


Alder Hey Hospital Eaton Road West Derby Liverpool Merseyside L12 2AP


Thursfield, Rebecca

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Barts Health NHS Trust


Trust headquarters 5th Floor Aneurin Bevan House 81 Commercial Road London E1 1RD



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Bristol Royal Hospital for Children


Upper Maudlin Street Bristol Avon BS2 8BJ



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Leeds Teaching Hospitals NHS Trust


St. James's University Hospital Beckett Street Leeds West Yorkshire LS9 7TF

Recruitment ends

December 2019



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