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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

Closed to recruitment - in follow up

Participating Centres
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 II

Full title

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. Primary Objectives 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


Trial type


Length of participation
the length of time a participant will take part in a trial, from the first to the last appointment

20 weeks

the name of the treatment or therapy being researched

CFTR Modulators

Recruitment target
the number of participants who need to be recruited for the trial in the UK


Last edited date

20 November 2017

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?


13 - 17

Top inclusion criteria
  • Stable CF disease as judged by the investigator.
  • Sweat chloride value ≥60 mmol/L from test results obtained during screening.
  • Heterozygous for F508del and a minimal function (MF) mutation known or predicted not to be responsive to TEZ and/or IVA.
Top exclusion criteria
  • History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
  • History of an acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug with portal hypertension.

CF centres running this trial


Heart of England NHS Foundation Trust


Birmingham Heartlands Hospital Bordesley Green East Birmingham West Midlands B9 5SS

Recruitment starts

February 2017

Recruitment ends

August 2017


Nash, Edward

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Royal Brompton and Harefield NHS Foundation Trust


Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment starts

February 2017

Recruitment ends

December 2017


Davies, Jane C

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Southampton General Hospital


Tremona Road Southampton Hampshire SO16 6YD

Recruitment starts

February 2017

Recruitment ends

November 2017


Carroll, Mary

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