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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)

Details

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

Open to recruitment

Participating Centres
Phase
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

124417

Trial type

Medication

Intervention
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

250

Last edited date

06 January 2020

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type

Commercial

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

CF centres running this trial

Recruiting

ST JAMES'S UNIVERSITY HOSPITAL

Address

BECKETT STREET, LEEDS, WEST YORKSHIRE LS9 7TF

Recruitment starts

November 2019

Recruitment ends

July 2020

Contact

Peckham, Daniel

Get in touch

Closed

Queen Elizabeth University Hospital

Address

1345 Govan Road G51 4TF

Recruitment starts

November 2019

Contact

MacGregor, Gordon

Get in touch

Closed

Royal Brompton Hospital

Address

Sydney Street London SW3 6NP

Recruitment starts

December 2019

Recruitment ends

March 2020

Contact

Davies, Jane C

Get in touch

Closed

Royal Devon and Exeter Hospital (Wonford)

Address

Royal Devon and Exeter Hospital Barrack Road Exeter Devon EX2 5DW

Recruitment starts

November 2019

Recruitment ends

February 2020

Contact

Withers, Nick

Get in touch
Show all participating centres