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A study to learn more about a combination of CFTR modulator medicines in people with Cystic Fibrosis (VX 18 445 104)


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

Project in Setup

the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase III

Full title

A study to evaluate the effectiveness and safety of VX 445/Tezacaftor/Ivacaftor in people with Cystic Fibrosis who are heterozygous for the F508del mutation and a gating or residual function mutation

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 heterozygous for the F508del mutation and a gating or residual function 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 9 times over a 5 month period where a study assessment 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

22 October 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?

Top inclusion criteria
  • Heterozygous for F508del and either a gating or residual function mutation
  • 12 Years and older
  • FEV1 value ≥40% and ≤90%
Top exclusion criteria
  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection