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
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
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
Add to watchlist
the name of the treatment or therapy being researched
the number of participants who need to be recruited for the trial in the UK
Last edited date
06 January 2020
Vertex Pharmaceuticals Incorporated
CF sponsor type