Your donation will make a difference:
Cystic Fibrosis Trust

Please Log in

Log in

Not a registered user? Register with us

This trial has been added to your watchlist.

Do you want to receive email notifications when the trial status changes?

No Yes

Unfollowing a trial will remove it from the watchlist and stop status change email notifications

Are you sure you want to unfollow this trial?

Yes, remove from watchlist

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor


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


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 III

Full title

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Longterm Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508delCFTR Mutation

This study will look at whether two drugs (called lumacaftor and ivacaftor) can help treat cystic fibrosis when taken together over a long period of time. It will also look at whether they are safe over this period. You can only take part in this study if you took part in a study called VX14-809-109, and if you meet certain criteria. These criteria will be checked by the study doctor and nurse at a screening visit. If you meet these criteria you will be treated with lumacaftor and ivacaftor together for approximately 96 weeks. A follow up visit will be held 28 days after the last dose of lumacaftor and ivacaftor. You will need to attend the clinic for 15 visits over the study duration and will undergo a range of tests. Participants who stopped taking the study drugs early in the VX14-809-109 study will be invited to take part in an observational study. They will not receive the study drug but will receive regular phone calls.

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

96 weeks

the name of the treatment or therapy being researched

CFTR Modulators (Ivacaftor, Lumacaftor)

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


Last edited date

01 July 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?


0 - 12

Top inclusion criteria
  • Homozygous for the F508del-CFTR mutation
  • Weight >= 15Kg

CF centres running this trial


Leeds Teaching Hospitals NHS Trust


St. James's University Hospital Beckett Street Leeds West Yorkshire LS9 7TF

Recruitment starts

June 2016

Recruitment ends

March 2018


Lee, Timothy

Get in touch


Royal Brompton and Harefield NHS Foundation Trust


Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment starts

April 2016

Recruitment ends

June 2018


Davies, Jane C

Get in touch


Wythenshawe Hospital Manchester University NHS Foundation Trust


Southmoor Road Wythenshawe Manchester Greater Manchester M23 9LT


Barry, Peter

Get in touch