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 Phase 2, Randomized, Double-blind, Controlled Study to
Evaluate the Safety and Efficacy of VX-440 Monotherapy
and Combination Therapy in Subjects Aged 12 Years and
Older With Cystic Fibrosis
Cystic Fibrosis (CF) is a genetic disease caused by mutations in the gene encoding a cell transport protein (ion channel). This ion channel transports chloride ions in and out of cells in multiple organ systems. When ions are not transported correctly the salt and water balance in cells and tissues is not controlled, leading to the production of sticky mucus in the lungs, airways and intestines.
Vertex is developing treatments to help increasing production of the ion channel and increase transport of chloride ions. IVA and Orkambi are two drugs that have been approved for treatment. Vertex is also developing TEZ and now VX-440. VX-440 increases the production of the ion channel, but in a different way to the current treatments. So, it is hoped that by using VX-440 as well as current treatments the production of the ion channel will be increased even further.
1) To evaluate the safety and tolerability of VX-440 in dual and triple combination with tezacaftor (TEZ) and/or ivacaftor (IVA).
2) To evaluate to effectiveness of VX-440 in dual and triple combination with TEZ and/or IVA
Trial Reference Number
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Length of participation
the length of time a participant will take part in a trial, from the first to the last appointment
the name of the treatment or therapy being researched
Drug : Tezacaftor, Drug : Ivacaftor, Drug : VX-440, Drug : Matched Placebos
the number of participants who need to be recruited for the trial in the UK
Last edited date
20 November 2017
Vertex Pharmaceuticals Incorporated
CF sponsor type